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Category Archives: Human Genetic Engineering

Genetic Engineering in Humans – Curing Diseases and …

Over the past few years, the field of biotechnology has advanced at a very high rate that scientists can now edit plants and animals at the genomic level. Different genetic engineering or genome-editing techniques such aszinc fingernucleases, transcription activator-like effector nucleases (TALENs), meganucleases and theCRISPR/Cas9 system have aided scientists to alter genomes to create modified organisms.

Like in plants and animals, could genome-editing be performed in humans? Yes. But a bigger question arises here, should genome editing techniques be used to create designer babies, to remove heritable diseases or to enhance the human capabilities? It is one of the most controversial topics among scientists and hence it all comes down to ethics.

In a recent research, Shoukhrat Mitalipov of Oregon Health Sciences University in Portland reported successfully repairing a genetic mutation in human embryos bringing the idea of genetic engineering in humans closer to reality.

To understand the ethical implications of genetic engineering in humans, it is important to first understand the basics.

Genetic engineering is basically manipulating or changing the DNA to alter the organisms appearance in a particular way. The human body cells contain encoded information compiled into a form called genes, which are responsible for the bodys growth, structure and functioning. Human genetic engineering decodes this information and applies it to the welfare of mankind.

For example, all over the world, several scientists have reported the singing in mice. However, the frequencies at which they sing is not audible to humans. The Alstons brown mouse or Alstons singing mouse is a famous example. It would be interesting to hear these songs too.

Japanese geneticists at the University of Osaka were conducting a research to study the mutagenic effects in a strain of mice that were genetically engineered. Among many effects, the mutation may have caused the alteration in the vocalization in the mice giving birth to an offspring which could sing at a frequency audible to humans.This genetic modification (which was actually an accident) may help in studying the communication patterns in mice as well as in comparing of similarities and differences with other mammals. Some other examples of genetic engineering are GloFish, drug-producing chickens, cows that make human-like milk, diesel-producing bacteria, banana vaccines and disease-preventing mosquitoes.

Based on their type of cell, there are two types of genetic engineering;

Human genetic engineering can further be classified into two types;

In human genetic engineering, the genes or the DNA of a person is changed. This can be used to bring about structural changes in human beings. More importantly, it can be used to introduce the genes for certain positive and desirable traits in embryos. Genetic engineering in humans can result in finding a permanent cure for many diseases.

Some people are born with or acquire exceptional qualities. If the genes responsible for these qualities can be identified, they can be introduced in the early embryos. The embryo develops into a baby called Designer baby or customized baby. Human genetic engineering is advancing at an increasing rate and might evolve to such an extent discovering new genes and implanting them into human embryos will be possible.

Let us take an example of bacteria to understand how genetic engineering works. Insulin is aprotein produced in the pancreasthat helps in the regulation of the sugar levels in our blood. People with type 1diabetes eithercannot produce insulin or produce insufficient insulin in the body. They have to acquire insulin from external sources to control their blood sugar levels. In 1982, Genetic engineering was used to produce a type of insulin which is similar to the human insulin, called the Humulin frombacteria which was then approved and licensed for human use.

An illustration showing how genetic engineering is used to produce insulin in bacteriaCourtesy: Genome Research Limited

Using this process, Chinese scientists have edited the genome of the human embryo for the first time. According to Nature News report, Researchers at Sun Yat-sen University in Guangzhou, China, were partially successful in using a genetic engineering technique to modify a gene in non-viable human embryos which was responsible for the fatal blood disorder.

The technique used, called CRISPR (short for clustered regularly interspaced short palindromic repeats) technology involves an enzyme complex known as CRISPR/Cas9, originating in bacteria as a defence system. CRISPR is a short, repeated DNA sequence that matches the genetic sequence of interest to be modified by the researchers. CRISPR works along with the Cas9 enzyme that acts like molecular scissors and cuts the DNA at a specific site.

As explained by John Reidhaar-Olson, a biochemist at Albert Einstein College of Medicine in New York First, in a simple explanation, the CRISPR/Cas9 complex navigates through the cells DNA, searching for the sequence that matches the CRISPR and binds to the sequence once found. The Cas9 then cuts the DNA which, in this case, is repaired by inserting a piece of DNA desired by the researcher.

Since 2013, CRISPR system has been to edit genes in adult human cells and animal embryos but for the first time has been used for modification in human embryos.

Junjiu Huang, a genetics researcher at Sun Yat-sen University, injected the CRISPR/Cas9 complex into human embryos with the aim of repairing a gene responsible for Beta thalassaemia which is a fatal blood disorder that reduces the production of haemoglobin. The non-viable embryos were obtained from local fertility clinics. These embryos would have been unable to survive independently after birth or develop properly as they had been fertilized by two sperms. The procedure was performed on 86 embryos and gene editing was allowed to take place in four days. Out of 86, 71 of the embryos survived and 54 of them were tested.

Splicing (removal of introns and joining of exonsineukaryotic mRNA) only occurred in 28 embryos successfully indicating the removal of faulty gene and the incorporation of the healthy gene in its place. However, in order for the technique to be used in viable human embryos, the success rate would need to be closer to 100%.

While partial success was achieved, certain worrisome mutations responsible for the detrimental effect on cells during gene-editing were also observed and at a much higher rate in mouse embryos or adult human cells undergoing the same procedure.

One of the most beneficial applications of genetic engineering is gene therapy. Gene therapy is one of the most important benefits of human genetic engineering. Over the last few years, gene therapy has successfully treated certain heart diseases. Driven by this success, researchers are working to find cures for all the genetic diseases. This will eventually lead to a healthier and more evolved human race.Inspired by the recent success of gene therapy trialsin human children and infants, researchers are now moving towards the treatment of genetic disorders before birth. The idea of using fetal gene therapy to treat genetic disorders that cant be treated after birth has generated hype among some of the scientists. Parents will be able to look forward to a healthy baby. Genetic engineering can be done in embryos prior to implantation into the mother.However, some are also questioning the feasibility and practicality of the therapy in humans.

While genetic engineering or modification may seem easy to cure diseases, it may produce certain side effects. While focusing on and treating one defect, there is a possibility it may cause another. A cell is responsible for various functions in the body and manipulating its genes without any counter effect or side effect may not be that easy.

Other than side effects, Cloning, for instance, can lead to an ethical disturbance among the humans risking the individuality and the diversity of human beings. Ironically, man will become just another man-made thing!

Among the social aspects of human genetic engineering, it can impose a heavy financial burden on the society, which may cause a rift between the rich and the poor in the society. Its feasibility and most importantly its affordability will also be a determinant of its popularity.

Human genetic engineering is a widely and rapidly advancing field. It can lead to miracles. But when assessing its benefits, its threats need to be assessed carefully too. Human genetic engineering can be beneficial to human beings and its potential advantages can come into reality only if it is handled with responsibility.

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Pros and Cons of Genetic Engineering – Conserve Energy Future

Genetic engineering is the process to alter the structure and nature of genes in human beings, animals or foods using techniques like molecular cloning and transformation. In other words, it is the process of adding or modifying DNA in an organism to bring about great deal of transformation.

Genetic engineering was thought to be a real problem just a few short years ago. We feared that soon we would be interfering with nature, trying to play God and cheat him out of his chance to decide whether we were blonde or dark haired, whether we had blue or bright green eyes or even how intelligent we were. The queries and concerns that we have regarding such an intriguing part of science are still alive and well, although they are less talked about nowadays than they were those few years ago.

However, this does not mean that they are any less relevant. In fact, they are as relevant today as they ever were. There are a number of very real and very troubling concerns surrounding genetic engineering, although there are also some very real benefits to further genetic engineering and genetic research, too. It seems, therefore, as though genetic engineering is both a blessing and a curse, as though we stand to benefit as well as lose from developing this area of science even further.

With genetic engineering, we will be able to increase the complexity of our DNA, and improve the human race. But it will be a slow process, because one will have to wait about 18 years to see the effect of changes to the genetic code.Stephen Hawking

Although at first the pros of genetic engineering may not be as apparent as the cons, upon further inspection, there are a number of benefits that we can only get if scientists consider to study and advance this particular branch of study. Here are just a few of the benefits:

1. Tackling and Defeating Diseases

Some of the most deadly and difficult diseases in the world, that have so resisted destruction, could be wiped out by the use of genetic engineering. There are a number of genetic mutations that humans can suffer from that will probably never be ended unless we actively intervene and genetically engineer the next generation to withstand these problems.

For instance, Cystic Fibrosis, a progressive and dangerous disease for which there is no known cure, could be completely cured with the help of selective genetic engineering.

2. Getting Rid of All Illnesses in Young and Unborn Children

There are very many problems that we can detect even before children are born. In the womb, doctors can tell whether your baby is going to suffer from sickle cell anemia, for instance, or from Down s syndrome. In fact, the date by which you can have an abortion has been pushed back relatively late just so that people can decide whether or not to abort a baby if it has one or more of these sorts of issues.

However, with genetic engineering, we would no longer have to worry. One of the main benefit of genetic engineering is that it can help cure and diseases and illness in unborn children. All children would be able to be born healthy and strong with no diseases or illnesses present at birth. Genetic engineering can also be used to help people who risk passing on terribly degenerative diseases to their children.

For instance, if you have Huntingtons there is a 50% chance that your children with inherit the disease and, even if they do not, they are likely to be carriers of the disease. You cannot simply stop people from having children if they suffer from a disease like this, therefore genetic engineering can help to ensure that their children live long and healthy lives from either the disease itself or from carrying the disease to pass on to younger generations.

3. Potential to Live Longer

Although humans are already living longer and longer in fact, our lifespan has shot up by a number of years in a very short amount of time because of the advances of modern medical science, genetic engineering could make our time on Earth even longer. There are specific, common illnesses and diseases that can take hold later in life and can end up killing us earlier than necessary.

With genetic engineering, on the other hand, we could reverse some of the most basic reasons for the bodys natural decline on a cellular level, drastically improving both the span of our lives and the quality of life later on. It could also help humans adapt to the growing problems of, for instance, global warming in the world.

If the places we live in become either a lot hotter or colder, we are going to need to adapt, but evolution takes many thousands of years, so genetic engineering can help us adapt quicker and better.

4. Produce New Foods

Genetic engineering is not just good for people. With genetic engineering we can design foods that are better able to withstand harsh temperatures such as the very hot or very cold, for instance and that are packed full of all the right nutrients that humans and animals need to survive. We may also be able to make our foods have a better medicinal value, thus introducing edible vaccines readily available to people all over the world

Perhaps more obvious than the pros of genetic engineering, there are a number of disadvantages to allowing scientists to break down barriers that perhaps are better left untouched. Here are just a few of those disadvantages:

1. Is it Right?

When genetic engineering first became possible, peoples first reactions were to immediately question whether it was right? Many religions believe that genetic engineering, after all, is tantamount to playing God, and expressly forbid that it is performed on their children, for instance.

Besides the religious arguments, however, there are a number of ethic objections. These diseases, after all, exist for a reason and have persisted throughout history for a reason. Whilst we should be fighting against them, we do need at least a few illnesses, otherwise we would soon become overpopulated. In fact, living longer is already causing social problems in the world today, so to artificially extend everybodys time on Earth might cause even more problems further down the line, problems that we cannot possibly predict.

2. May Lead to Genetic Defects

Another real problem with genetic engineering is the question about the safety of making changes at the cellular level. Scientists do not yet know absolutely everything about the way that the human body works (although they do, of course, have a very good idea). How can they possibly understand the ramifications of slight changes made at the smallest level?

What if we manage to wipe out one disease only to introduce something brand new and even more dangerous? Additionally, if scientists genetically engineer babies still in the womb, there is a very real and present danger that this could lead to complications, including miscarriage (early on), premature birth or even stillbirth, all of which are unthinkable.

The success rate of genetic experiments leaves a lot to be desired, after all. The human body is so complicated that scientists have to be able to predict what sort of affects their actions will have, and they simply cannot account for everything that could go wrong.

3. Limits Genetic Diversity

We need diversity in all species of animals. By genetically engineering our species, however, we will be having a detrimental effect on our genetic diversity in the same way as something like cloning would. Gene therapy is available only to the very rich and elite, which means that traits that tend to make people earn less money would eventually die out.

4. Can it Go Too Far?

One pressing question and issue with genetic engineering that has been around for years and years is whether it could end up going too far. There are many thousands of genetic scientists with honest intentions who want to bring an end to the worst diseases and illnesses of the current century and who are trying to do so by using genetic engineering.

However, what is to stop just a handful of people taking the research too far? What if we start demanding designer babies, children whose hair color, eye color, height and intelligence we ourselves dictate? What if we end up engineering the sex of the baby, for instance in China, where is it much more preferable to have a boy? Is that right? Is it fair? The problems with genetic engineering going too far are and ever present worry in a world in which genetic engineering is progressing further and further every day.

Genetic engineering is one of the topic that causes a lot of controversy. Altering the DNA of organisms has certainly raised a few eyebrows. It may work wonders but who knows if playing with the nature is really safe? Making yourself aware of all aspects of genetic engineering can help you to form your own opinion.

A true environmentalist by heart . Founded Conserve Energy Future with the sole motto of providing helpful information related to our rapidly depleting environment. Unless you strongly believe in Elon Musks idea of making Mars as another habitable planet, do remember that there really is no 'Planet B' in this whole universe.

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CRISPR: A game-changing genetic engineering technique …

Have you heard? A revolution has seized the scientific community. Within only a few years, research labs worldwide have adopted a new technology that facilitates making specific changes in the DNA of humans, other animals, and plants. Compared to previous techniques for modifying DNA, this new approach is much faster and easier. This technology is referred to as CRISPR, and it has changed not only the way basic research is conducted, but also the way we can now think about treating diseases [1,2].

CRISPR is an acronym for Clustered Regularly Interspaced Short Palindromic Repeat. This name refers to the unique organization of short, partially palindromic repeated DNA sequences found in the genomes of bacteria and other microorganisms. While seemingly innocuous, CRISPR sequences are a crucial component of the immune systems [3] of these simple life forms. The immune system is responsible for protecting an organisms health and well-being. Just like us, bacterial cells can be invaded by viruses, which are small, infectious agents. If a viral infection threatens a bacterial cell, the CRISPR immune system can thwart the attack by destroying the genome of the invading virus [4]. The genome of the virus includes genetic material that is necessary for the virus to continue replicating. Thus, by destroying the viral genome, the CRISPR immune system protects bacteria from ongoing viral infection.

Figure 1 ~ The steps of CRISPR-mediated immunity. CRISPRs are regions in the bacterial genome that help defend against invading viruses. These regions are composed of short DNA repeats (black diamonds) and spacers (colored boxes). When a previously unseen virus infects a bacterium, a new spacer derived from the virus is incorporated amongst existing spacers. The CRISPR sequence is transcribed and processed to generate short CRISPR RNA molecules. The CRISPR RNA associates with and guides bacterial molecular machinery to a matching target sequence in the invading virus. The molecular machinery cuts up and destroys the invading viral genome. Figure adapted from Molecular Cell 54, April 24, 2014 [5].

Interspersed between the short DNA repeats of bacterial CRISPRs are similarly short variable sequences called spacers (FIGURE 1). These spacers are derived from DNA of viruses that have previously attacked the host bacterium [3]. Hence, spacers serve as a genetic memory of previous infections. If another infection by the same virus should occur, the CRISPR defense system will cut up any viral DNA sequence matching the spacer sequence and thus protect the bacterium from viral attack. If a previously unseen virus attacks, a new spacer is made and added to the chain of spacers and repeats.

The CRISPR immune system works to protect bacteria from repeated viral attack via three basic steps [5]:

Step 1) Adaptation DNA from an invading virus is processed into short segments that are inserted into the CRISPR sequence as new spacers.

Step 2) Production of CRISPR RNA CRISPR repeats and spacers in the bacterial DNA undergo transcription, the process of copying DNA into RNA (ribonucleic acid). Unlike the double-chain helix structure of DNA, the resulting RNA is a single-chain molecule. This RNA chain is cut into short pieces called CRISPR RNAs.

Step 3) Targeting CRISPR RNAs guide bacterial molecular machinery to destroy the viral material. Because CRISPR RNA sequences are copied from the viral DNA sequences acquired during adaptation, they are exact matches to the viral genome and thus serve as excellent guides.

The specificity of CRISPR-based immunity in recognizing and destroying invading viruses is not just useful for bacteria. Creative applications of this primitive yet elegant defense system have emerged in disciplines as diverse as industry, basic research, and medicine.

In Industry

The inherent functions of the CRISPR system are advantageous for industrial processes that utilize bacterial cultures. CRISPR-based immunity can be employed to make these cultures more resistant to viral attack, which would otherwise impede productivity. In fact, the original discovery of CRISPR immunity came from researchers at Danisco, a company in the food production industry [2,3]. Danisco scientists were studying a bacterium called Streptococcus thermophilus, which is used to make yogurts and cheeses. Certain viruses can infect this bacterium and damage the quality or quantity of the food. It was discovered that CRISPR sequences equipped S. thermophilus with immunity against such viral attack. Expanding beyond S. thermophilus to other useful bacteria, manufacturers can apply the same principles to improve culture sustainability and lifespan.

In the Lab

Beyond applications encompassing bacterial immune defenses, scientists have learned how to harness CRISPR technology in the lab [6] to make precise changes in the genes of organisms as diverse as fruit flies, fish, mice, plants and even human cells. Genes are defined by their specific sequences, which provide instructions on how to build and maintain an organisms cells. A change in the sequence of even one gene can significantly affect the biology of the cell and in turn may affect the health of an organism. CRISPR techniques allow scientists to modify specific genes while sparing all others, thus clarifying the association between a given gene and its consequence to the organism.

Rather than relying on bacteria to generate CRISPR RNAs, scientists first design and synthesize short RNA molecules that match a specific DNA sequencefor example, in a human cell. Then, like in the targeting step of the bacterial system, this guide RNA shuttles molecular machinery to the intended DNA target. Once localized to the DNA region of interest, the molecular machinery can silence a gene or even change the sequence of a gene (Figure 2)! This type of gene editing can be likened to editing a sentence with a word processor to delete words or correct spelling mistakes. One important application of such technology is to facilitate making animal models with precise genetic changes to study the progress and treatment of human diseases.

Figure 2 ~ Gene silencing and editing with CRISPR. Guide RNA designed to match the DNA region of interest directs molecular machinery to cut both strands of the targeted DNA. During gene silencing, the cell attempts to repair the broken DNA, but often does so with errors that disrupt the geneeffectively silencing it. For gene editing, a repair template with a specified change in sequence is added to the cell and incorporated into the DNA during the repair process. The targeted DNA is now altered to carry this new sequence.

In Medicine

With early successes in the lab, many are looking toward medical applications of CRISPR technology. One application is for the treatment of genetic diseases. The first evidence that CRISPR can be used to correct a mutant gene and reverse disease symptoms in a living animal was published earlier this year [7]. By replacing the mutant form of a gene with its correct sequence in adult mice, researchers demonstrated a cure for a rare liver disorder that could be achieved with a single treatment. In addition to treating heritable diseases, CRISPR can be used in the realm of infectious diseases, possibly providing a way to make more specific antibiotics that target only disease-causing bacterial strains while sparing beneficial bacteria [8]. A recent SITN Waves article discusses how this technique was also used to make white blood cells resistant to HIV infection [9].

Of course, any new technology takes some time to understand and perfect. It will be important to verify that a particular guide RNA is specific for its target gene, so that the CRISPR system does not mistakenly attack other genes. It will also be important to find a way to deliver CRISPR therapies into the body before they can become widely used in medicine. Although a lot remains to be discovered, there is no doubt that CRISPR has become a valuable tool in research. In fact, there is enough excitement in the field to warrant the launch of several Biotech start-ups that hope to use CRISPR-inspired technology to treat human diseases [8].

Ekaterina Pak is a Ph.D. student in the Biological and Biomedical Sciences program at Harvard Medical School.

1. Palca, J. A CRISPR way to fix faulty genes. (26 June 2014) NPR < http://www.npr.org/blogs/health/2014/06/26/325213397/a-crispr-way-to-fix-faulty-genes> [29 June 2014]

2. Pennisi, E. The CRISPR Craze. (2013) Science, 341 (6148): 833-836.

3. Barrangou, R., Fremaux, C., Deveau, H., Richards, M., Boyaval, P., Moineau, S., Romero, D.A., and Horvath, P. (2007). CRISPR provides acquired resistance against viruses in prokaryotes. Science 315, 17091712.

4. Brouns, S.J., Jore, M.M., Lundgren, M., Westra, E.R., Slijkhuis, R.J., Snijders, A.P., Dickman, M.J., Makarova, K.S., Koonin, E.V., and van der Oost, J. (2008). Small CRISPR RNAs guide antiviral defense in prokaryotes. Science 321, 960964.

5. Barrangou, R. and Marraffini, L. CRISPR-Cas Systems: Prokaryotes Upgrade to Adaptive Immunity (2014). Molecular Cell 54, 234-244.

6. Jinkek, M. et al. A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity. (2012) 337(6096):816-21.

7. CRISPR reverses disease symptoms in living animals for first time. (31 March 2014). Genetic Engineering and Biotechnology News. <http://www.genengnews.com/gen-news-highlights/crispr-reverses-disease-symptoms-in-living-animals-for-first-time/81249682/> [27 July 2014]

8. Pollack, A. A powerful new way to edit DNA. (3 March 2014). NYTimes < http://www.nytimes.com/2014/03/04/health/a-powerful-new-way-to-edit-dna.html?_r=0> [16 July 2014]

9. Gene editing technique allows for HIV resistance? <http://sitn.hms.harvard.edu/flash/waves/2014/gene-editing-technique-allows-for-hiv-resistance/> [13 June 2014]

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Human Genetic Engineering – Probe Ministries

Although much has occurred in this field since this article was written in 2000, the questions addressed by Dr. Bohlin are still timely and relevant. Is manipulating our genetic code simply a tool or does it deal with deeper issues? Dealing with genetic engineering must be done within the context of the broader ethical and theological issues involved. In the article, Dr. Bohlin provides an excellent summary driven from his biblical worldview perspective.

Genetic technology harbors the potential to change the human species forever. The soon to be completed Human Genome Project will empower genetic scientists with a human biological instruction book. The genes in all our cells contain the code for proteins that provide the structure and function to all our tissues and organs. Knowing this complete code will open new horizons for treating and perhaps curing diseases that have remained mysteries for millennia. But along with the commendable and compassionate use of genetic technology comes the specter of both shadowy purposes and malevolent aims.

For some, the potential for misuse is reason enough for closing the door completelythe benefits just arent worth the risks. In this article, Id like to explore the application of genetic technology to human beings and apply biblical wisdom to the eventual ethical quagmires that are not very far away. In this section well investigate the various ways humans can be engineered.

Since we have introduced foreign genes into the embryos of mice, cows, sheep, and pigs for years, theres no technological reason to suggest that it cant be done in humans too. Currently, there are two ways of pursuing gene transfer. One is simply to attempt to alleviate the symptoms of a genetic disease. This entails gene therapy, attempting to transfer the normal gene into only those tissues most affected by the disease. For instance, bronchial infections are the major cause of early death for patients with cystic fibrosis (CF). The lungs of CF patients produce thick mucus that provides a great growth medium for bacteria and viruses. If the normal gene can be inserted in to the cells of the lungs, perhaps both the quality and quantity of their life can be enhanced. But this is not a complete cure and they will still pass the CF gene on to their children.

In order to cure a genetic illness, the defective gene must be replaced throughout the body. If the genetic defect is detected in an early embryo, its possible to add the gene at this stage, allowing the normal gene to be present in all tissues including reproductive tissues. This technique has been used to add foreign genes to mice, sheep, pigs, and cows.

However, at present, no laboratory is known to be attempting this well-developed technology in humans. Princeton molecular biologist Lee Silver offers two reasons.{1} First, even in animals, it only works 50% of the time. Second, even when successful, about 5% of the time, the new gene gets placed in the middle of an existing gene, creating a new mutation. Currently these odds are not acceptable to scientists and especially potential clients hoping for genetic engineering of their offspring. But these are only problems of technique. Its reasonable to assume that these difficulties can be overcome with further research.

The primary use for human genetic engineering concerns the curing of genetic disease. But even this should be approached cautiously. Certainly within a Christian worldview, relieving suffering wherever possible is to walk in Jesus footsteps. But what diseases? How far should our ability to interfere in life be allowed to go? So far gene therapy is primarily tested for debilitating and ultimately fatal diseases such as cystic fibrosis.

The first gene therapy trial in humans corrected a life-threatening immune disorder in a two-year-old girl who, now ten years later, is doing well. The gene therapy required dozens of applications but has saved the family from a $60,000 per year bill for necessary drug treatment without the gene therapy.{2} Recently, sixteen heart disease patients, who were literally waiting for death, received a solution containing copies of a gene that triggers blood vessel growth by injection straight into the heart. By growing new blood vessels around clogged arteries, all sixteen showed improvement and six were completely relieved of pain.

In each of these cases, gene therapy was performed as a last resort for a fatal condition. This seems to easily fall within the medical boundaries of seeking to cure while at the same time causing no harm. The problem will arise when gene therapy will be sought to alleviate a condition that is less than life-threatening and perhaps considered by some to simply be one of lifes inconveniences, such as a gene that may offer resistance to AIDS or may enhance memory. Such genes are known now and many are suggesting that these goals will and should be available for gene therapy.

The most troublesome aspect of gene therapy has been determining the best method of delivering the gene to the right cells and enticing them to incorporate the gene into the cells chromosomes. Most researchers have used crippled forms of viruses that naturally incorporate their genes into cells. The entire field of gene therapy was dealt a severe setback in September 1999 upon the death of Jesse Gelsinger who had undergone gene therapy for an inherited enzyme deficiency at the University of Pennsylvania.{3} Jesse apparently suffered a severe immune reaction and died four days after being injected with the engineered virus.

The same virus vector had been used safely in thousands of other trials, but in this case, after releasing stacks of clinical data and answering questions for two days, the researchers didnt fully understand what had gone wrong.{4} Other institutions were also found to have failed to file immediate reports as required of serious adverse events in their trials, prompting a congressional review.{5} All this should indicate that the answers to the technical problems of gene therapy have not been answered and progress will be slowed as guidelines and reporting procedures are studied and reevaluated.

The simple answer is no, at least for the foreseeable future. Gene therapy currently targets existing tissue in a existing child or adult. This may alleviate or eliminate symptoms in that individual, but will not affect future children. To accomplish a correction for future generations, gene therapy would need to target the germ cells, the sperm and egg. This poses numerous technical problems at the present time. There is also a very real concern about making genetic decisions for future generations without their consent.

Some would seek to get around these difficulties by performing gene therapy in early embryos before tissue differentiation has taken place. This would allow the new gene to be incorporated into all tissues, including reproductive organs. However, this process does nothing to alleviate the condition of those already suffering from genetic disease. Also, as mentioned earlier this week, this procedure would put embryos at unacceptable risk due to the inherent rate of failure and potential damage to the embryo.

Another way to affect germ line gene therapy would involve a combination of gene therapy and cloning.{6} An embryo, fertilized in vitro, from the sperm and egg of a couple at risk for sickle-cell anemia, for example, could be tested for the sickle-cell gene. If the embryo tests positive, cells could be removed from this early embryo and grown in culture. Then the normal hemoglobin gene would be added to these cultured cells.

If the technique for human cloning could be perfected, then one of these cells could be cloned to create a new individual. If the cloning were successful, the resulting baby would be an identical twin of the original embryo, only with the sickle-cell gene replaced with the normal hemoglobin gene. This would result in a normal healthy baby. Unfortunately, the initial embryo was sacrificed to allow the engineering of its identical twin, an ethically unacceptable trade-off.

So what we have seen, is that even human gene therapy is not a long-term solution, but a temporary and individual one. But even in condoning the use of gene therapy for therapeutic ends, we need to be careful that those for whom gene therapy is unavailable either for ethical or monetary reasons, dont get pushed aside. It would be easy to shun those with uncorrected defects as less than desirable or even less than human. There is, indeed, much to think about.

The possibility of someone or some government utilizing the new tools of genetic engineering to create a superior race of humans must at least be considered. We need to emphasize, however, that we simply do not know what genetic factors determine popularly desired traits such as athletic ability, intelligence, appearance and personality. For sure, each of these has a significant component that may be available for genetic manipulation, but its safe to say that our knowledge of each of these traits is in its infancy.

Even as knowledge of these areas grows, other genetic qualities may prevent their engineering. So far, few genes have only a single application in the body. Most genes are found to have multiple effects, sometimes in different tissues. Therefore, to engineer a gene for enhancement of a particular traitsay memorymay inadvertently cause increased susceptibility to drug addiction.

But what if in the next 50 to 100 years, many of these unknowns can be anticipated and engineering for advantageous traits becomes possible. What can we expect? Our concern is that without a redirection of the worldview of the culture, there will be a growing propensity to want to take over the evolution of the human species. The many people see it, we are simply upright, large-brained apes. There is no such thing as an independent mind. Our mind becomes simply a physical construct of the brain. While the brain is certainly complicated and our level of understanding of its intricate machinery grows daily, some hope that in the future we may comprehend enough to change who and what we are as a species in order to meet the future demands of survival.

Edward O. Wilson, a Harvard entomologist, believes that we will soon be faced with difficult genetic dilemmas. Because of expected advances in gene therapy, we will not only be able to eliminate or at least alleviate genetic disease, we may be able to enhance certain human abilities such as mathematics or verbal ability. He says, Soon we must look deep within ourselves and decide what we wish to become.{7} As early as 1978, Wilson reflected on our eventual need to decide how human we wish to remain.{8}

Surprisingly, Wilson predicts that future generations will opt only for repair of disabling disease and stop short of genetic enhancements. His only rationale however, is a question. Why should a species give up the defining core of its existence, built by millions of years of biological trial and error?{9} Wilson is naively optimistic. There are loud voices already claiming that man can intentionally engineer our evolutionary future better than chance mutations and natural selection. The time to change the course of this slow train to destruction is now, not later.

Many of the questions surrounding the ethical use of genetic engineering practices are difficult to answer with a simple yes or no. This is one of them. The answer revolves around the method used to determine the sex selection and the timing of the selection itself.

For instance, if the sex of a fetus is determined and deemed undesirable, it can only be rectified by termination of the embryo or fetus, either in the lab or in the womb by abortion. There is every reason to prohibit this process. First, an innocent life has been sacrificed. The principle of the sanctity of human life demands that a new innocent life not be killed for any reason apart from saving the life of the mother. Second, even in this country where abortion is legal, one would hope that restrictions would be put in place to prevent the taking of a life simply because its the wrong sex.

However, procedures do exist that can separate sperm that carry the Y chromosome from those that carry the X chromosome. Eggs fertilized by sperm carrying the Y will be male, and eggs fertilized by sperm carrying the X will be female. If the sperm sample used to fertilize an egg has been selected for the Y chromosome, you simply increase the odds of having a boy (~90%) over a girl. So long as the couple is willing to accept either a boy or girl and will not discard the embryo or abort the baby if its the wrong sex, its difficult to say that such a procedure should be prohibited.

One reason to utilize this procedure is to reduce the risk of a sex-linked genetic disease. Color-blindness, hemophilia, and fragile X syndrome can be due to mutations on the X chromosome. Therefore, males (with only one X chromosome) are much more likely to suffer from these traits when either the mother is a carrier or the father is affected. (In females, the second X chromosome will usually carry the normal gene, masking the mutated gene on the other X chromosome.) Selecting for a girl by sperm selection greatly reduces the possibility of having a child with either of these genetic diseases. Again, its difficult to argue against the desire to reduce suffering when a life has not been forfeited.

But we must ask, is sex determination by sperm selection wise? A couple that already has a boy and simply wants a girl to balance their family, seems innocent enough. But why is this important? What fuels this desire? Its dangerous to take more and more control over our lives and leave the sovereignty of God far behind. This isnt a situation of life and death or even reducing suffering.

But while it may be difficult to find anything seriously wrong with sex selection, its also difficult to find anything good about it. Even when the purpose may be to avoid a sex-linked disease, we run the risk of communicating to others affected by these diseases that because they could have been avoided, their life is somehow less valuable. So while it may not be prudent to prohibit such practices, it certainly should not be approached casually either.

Notes

1. Lee Silver, Remaking Eden: Cloning and Beyond in a Brave New World, New York, NY: Avon Books, p. 230-231. 2. Leon Jaroff, Success stories, Time, 11 January 1999, p. 72-73. 3. Sally Lehrman, Virus treatment questioned after gene therapy death, Nature Vol. 401 (7 October 1999): 517-518. 4. Eliot Marshall, Gene therapy death prompts review of adenovirus vector, Science Vol. 286 (17 December 1999): 2244-2245. 5. Meredith Wadman, NIH under fire over gene-therapy trials, Nature Vol. 403 (20 January 1999): 237. 6. Steve Mirsky and John Rennie, What cloning means for gene therapy, Scientific American, June 1997, p. 122-123. 7. Ibid., p. 277. 8. Edward Wilson, On Human Nature, Cambridge, Mass.: Harvard University Press, p. 6. 9. E. Wilson, Consilience, p. 277.

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Chinese Scientist’s Human Genetic Engineering Experiment …

GREG WILPERT: Its The Real News Network and Im Greg Wilpert, coming to you from Baltimore.

A scientist in China made what should be a momentous announcement on Monday. He claimed to have successfully edited the genes of a pair of twins who were born earlier this month. The Chinese scientist He Jiankui said that he altered the twins genes so that they would be resistant to the HIV virus, using a gene editing technique known as CRISPR. Heres how he justified the project in an interview with The Associated Press.

HE JIANKUI: I feel a strong responsibility that its not just to make it the first, but also make it an example how to perform like this, consider morality of the society and consider its impact to the public.

GREG WILPERT: Genetic engineering of human genes is illegal in the United States and in most other countries with the potential technology to do so. However, in China, theres no law against it, even though many scientists have expressed strong opposition to the practice. Joining me now to discuss the implications of this announcement is Professor Stuart Newman. Hes professor of cell biology and anatomy at New York Medical College in Valhalla, and he is a founding member of the Council for Responsible Genetics and editor-in-chief of the journal Biological Theory. He is also the author of the forthcoming book, Biotech Juggernaut. Thanks, Stuart, for joining us today.

STUART NEWMAN: Thank you.

GREG WILPERT: So the scientist who did this, He Jiankui, he said that he succeeded in this engineering project, but he did not provide any proof that it actually worked. How likely do you think it is that it actually did work?

STUART NEWMAN: Well, I think hes a serious scientist. I wont comment right now on the morality of what he did, but I think that he knows what hes doing scientifically. And Ive met him, and I think that his claim, as far as I can tell, is probably valid.

GREG WILPERT: So in an article that you published last year, you expressed skepticism that the CRISPR technology could actually do some of this kind of genetic engineering that was used in this particular test. Why is it, what is the issue around well get to the morality later, but I just want to get into the technique for a second. Why are you skeptical about this project of genetic engineering using this kind of technique?

STUART NEWMAN: Well, theres a difference between modifying a gene, even accurately modifying a gene, and bringing about a phenotypic effect that has a biological effect. So in the article that you probably saw, I said that CRISPR wont be useful in bringing about the results that people want because the way genes operate in embryos is not the way that they operate in adult organisms. In an adult organism, you can look at a gene and say it more or less does one thing or it does two things. During embryonic development, it interacts with many other genes in a very quickly changing system and the proteins that the gene specifies dont necessarily do the same thing during development that they do in the adults. So I was skeptical about the ability to bring about desired results. But if its claimed that CRISPR can take a piece of DNA and change it in a specific way, yes it can do that.

GREG WILPERT: So as we saw in the clip of He Jiankui, he says that he felt it was important to do this and to do it for basically what he considered to be a good cause or a good reason. Whats your reaction to this argument and what do you see as being the dangers of this type of work?

STUART NEWMAN: I think its very unjustified that he did it. First of all, He is just looking at the known function of the gene in adult humans, hes not looking at the function during embryonic development. Theres a whole set of unknowns in the developmental process and we dont really have good scientific control over manipulating it, and we may never do because its so complicated. So He has taken a gene with a known function in the adult and hes said its bad to have that gene active, so he inactivated it. But really, theres lot of misconceptions, kind of unthinking, kind of moving ahead in what he did that he should have never done it.

GREG WILPERT: So what is at stake here, basically, and what do you see as being the best way to avoid the worst kinds of consequences of this technology?

STUART NEWMAN: Well I think you know hes taking something that I guess he would say everybody agrees it would be good to be resistant against, AIDS or other viral diseases. So hes looking for some kind of agreement in what he did by the particular problem that he addressed. But in fact, what some people consider an impairment other people dont consider an impairment. In particularly American society, I cant speak for Chinese society, theres a kind of a consumerist ethic which says that if somebody wants to pay for something and its possible to do, they should be allowed to do it.

And in fact, you said at the top of the segment that there are laws against it in the United States, but there really arent. There are not laws in the United States against genetically modifying embryos. So we would have to pass such laws in order to prevent it from happening. And even passing the laws wont prevent it from happening because therell people who do it surreptitiously. So I think that we really have to talk about it a lot. It has to be stigmatized it has to be something that a lot of opprobrium falls on somebody who would attempt such a thing because in many cases it will turn out badly. And then what do you do with one of the unfortunate outcomes that turned out worse rather than better than not and hoped for. This is really a totally poor and motivated project.

GREG WILPERT: Well, what do you think, first of all, are the motivations behind this technology and this project?

STUART NEWMAN: Well, its just kind of a simple-minded approach to a medical problem. I mean, its like saying that AIDS is bad, this gene is associated with AIDS, get rid of this gene and we wont have AIDS or something. So really, it wont affect AIDS and the population, it will affect it in a couple of individuals. And if those individuals that have been genetically modified if it works and I doubt that it will work as intended. But if even if it does work itll just give a license to those resistant individuals to act responsibly and not use precautions and get themselves tested if theyre at risk and so on. So its really crazy, actually, I would call it crazy to try to do this. And scientifically, its based on a poor understanding of science.

GREG WILPERT: I guess the main issue here, perhaps, is that theres a lot of potential for unintended consequences and that the biology is a lot more complicated than people make it out to be if you look only at the individual genes. Is that more or less it in a nutshell?

STUART NEWMAN: Thats absolutely true, yes. And theres a kind of a false notion that if you understand an organisms genes, if you can modify the organisms genes, you can understand how the organism works and you can get it to work in a new way, in kind of an engineering paradigm. And this is not true at all. Genes are not the only thing that are controlling what goes on in an organism, particularly during early development. There are many forces, there physical forces, environmental forces involved in molding the embryos, not just the genes. And the other thing thats not recognized in these attempts is that genes dont always do the same thing in the same context. So the very same gene acting at different stages in the life history of an individual can do very different things, and this is not taken into account at all in these experiments.

GREG WILPERT: OK. Well, well leave it there for now. I was speaking to Stuart Newman, professor of cell biology and anatomy at New York Medical College in Valhalla. Thanks again, Stuart, for having joined us today.

STUART NEWMAN: Thank you.

GREG WILPERT: And thank you for joining The Real News Network. If you like Real News Network stories such as this one, please keep in mind that we have started our winter fundraiser and need your help to reach our goal of raising four hundred thousand dollars. Every dollar that you donate will be matched. Unlike practically all other news outlets, we do not accept support from governments or corporations. Please do what you can today.

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Human Genetic Engineering | APNORC.org | APNORC.org

Americans favor the use of gene editing to prevent disease or disabilities, while there is strong opposition to using the technology to change a babys physical characteristics, such eye color or intelligence. Support for eradicating disease and disabilities was strong regardless of party identification, education or religious preference.The same holds true for the opposition to altering genes in order to change physical features or capabilities.

Americans hold similar views about the ethics of gene editing.About 6 in 10 consider editing the genes of embryos for the purpose of preventing or reducing the risk of disease to be morally acceptable.Fifty-four percent say using the technology to prevents a non-fatal condition such as blindness as morally acceptable.Two-thirds say it is morally unacceptable to use gene editing to change a babys physical features or characteristics.

What about altering an adults genetic material without changing the genes of their offspring?The idea of using gene editing technology to prevent or cure a genetic disorder in an adult is supported by 56 percent, opposed by 17 percent, and 27 percent neither favor nor oppose.

While Americans favor using gene editing to deal with physical ailments, there is less support for the use of taxpayer money to finance testing on human embryos to develop the technology. Overall, 48 percent oppose federal funding to test gene editing technology, while 26 percent favor it and 25 percent neither favor nor oppose. Republicans are particularly against using government money for the development of gene editing.

Regardless of support for the technology, there are some concerns about possible ramifications.Fifty-two percent say the unethical use of gene editing is very likely, and 45 percent think it's very likely the technology would have unintended effects on human evolution. Few think it's likely that most people would be able to afford the technology.

Most Americans say it is at least somewhat likely that the development of gene editing technology will lead to further medical advances, eliminate many genetic illnesses, and be adequately tested.

The nationwide poll was conducted December 13-16, 2018 using the AmeriSpeak Panel, the probability-based panel of NORC at the University of Chicago. Online and telephone interviews using landlines and cell phones were conducted with 1,067 adults. The margin of sampling error is plus or minus 4.1 percentage points.

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