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Category Archives: Human Genetic Engineering

Synthetic Biology Speeds the Creation of Lab-Grown Livers – India Education Diary

Researchers at the University of Pittsburgh School of Medicine have combined synthetic biology with a machine-learning algorithm to create human liver organoids with blood- and bile-handling systems. When implanted into mice with failing livers, the lab-grown replacement livers extended life.

The study, published today in Cell Systems, shows that its possible to trigger and speed up the maturation of a lab-grown organ without sacrificing precision or control.

Mo Ebrahimkhani lab featurePregnancy is nine monthsit takes that long and even months after birth for new organs to maturebut if a person needs a liver, they may not be able to wait that long, said study author Mo Ebrahimkhani, M.D., associate professor of pathology and bioengineering, and member of the Pittsburgh Liver Research Center and the McGowan Institute for Regenerative Medicine. We showed its possible to get human liver tissue with four main cell types and vasculature in 17 days. We can mature tissue almost to the third trimester in only three months.

Other groups have attempted to coax organoid maturation in a dish using growth factors, but its expensive, inconsistent and prone to human error, Ebrahimkhani said. Often, there are unwanted tissue or cell typessuch as intestine or brain cells growing in the middle of what should be solid liver.

Using genetic engineering is cleaner but also more complex to orchestrate. So, Ebrahimkhani partnered with Patrick Cahan, Ph.D., at Johns Hopkins University to use a machine-learning system that can reverse engineer the genes necessary for human liver maturation.

Ebrahimkhani Vasculature releaseThen, Ebrahimkhani together with his collaborator at Pitt, Samira Kiani, M.D., applied genetic engineering techniques, including CRISPR, to turn a mass of immature liver tissueoriginally derived from human stem cellsinto what the team calls designer liver organoids.

The more mature the organoids got, the more capillaries and rudimentary bile duct cells snaked their way through the thin sheet of tissue, and the more closely the function of the tiny organ rivaled its full-size natural human model. Energy storage, fat accumulation, chemical transport, enzyme activity and protein production were all closer to adult human liver function, though still not a perfect match.

Ebrahimkhani imagines designer organoids having three main uses: drug discovery, disease modeling and organ transplant. Since the stem cells can come from the patients own body, lab-grown organs could be personalized, so there would be no threat of immune rejection.

When transplanted into mice with damaged livers, Ebrahimkhanis designer liver organoids successfully integrated into the animals bodies and continued to workproducing human proteins that showed up in the animals blood and prolonging the animals lives.

This is a proof-of-principle to show that its possible, Ebrahimkhani said. The technique could potentially go much further.

Our reference was a nature-designed human liver, but you can go after any design you like. For instance, you can make a genetic switch that protects the tissue from a virus, target the DNA of the virus and destroy it, Ebrahimkhani said. That sets this method apart.

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Hamsters genetically engineered by USU researchers are on the front lines of COVID-19 vaccine trials in Belgium –

LOGAN Genetically engineered golden Syrian hamsters developed by Utah State University researchers played a key role in animal trials of a possible vaccine to protect against the virus that causes COVID-19.

The Rega Institute in Leuven, Belgium, has used the hamsters produced by professor Zhongde Wang and his lab at USU to test the safety and effectiveness of a possible vaccine.

Details of the research conducted by the Rega Institute and its findings were published online in the journal Nature this week.

The candidate vaccine was found to be safe and effective in several animal models by a team of scientists at the institute.

Animal models play a vital role in vaccine research "because we cannot directly test them in humans. We need to use animal models, (it's) very critical," Wang said.

Wang said two pairs of hamsters were shipped to the Belgium lab in 2018 to start a breeding colony in an agreement with his lab.

"The scientists in my lab and I are very gratified that our research is contributing to combating this raging COVID-19 pandemic," Wang said in a statement.

"We also feel grateful for the excellent support from USU's Laboratory Animal Research Center to help us to carry out the research."

The Wang lab, established at USU in 2012, developed the first genetic hamster models in the world. The models are used in more than a dozen labs and institutions including the National Institutes of Health, the U.S. Army Medical Research Institute of Infectious Diseases, and Public Health Agency of Canada.

Hamsters from Wang's lab are also utilized in COVID-19 and other studies in USU's Institute for Antiviral Research.

"We pioneered development of genetic engineering techniques in this species and now we have about 30 different models. These are 30 different genetic modifications," Wang said in an interview Wednesday,

Typically, rodents carry many disease-causing organisms without becoming sick. The USU lab genetically engineered the golden Syrian hamsters to be susceptible to viruses that infect humans.

Viruses frequently attach to receptors in humans that are not present in animals, which limits effective testing of potential drugs to prevent or treat diseases. Hamsters from Wang's lab have a human gene inserted into their DNA for the receptor to which this coronavirus binds to facilitate testing, according to a university press release.

Because the hamsters are designed specifically to react to disease challenges more like humans, it takes fewer experiments to verify results, which expedites the process and can reduce numbers of animals used in research.

"We take animal welfare extremely seriously, and only the minimum numbers of animals required are used," said Wang, a professor in the Department of Animal, Dairy and Veterinary Sciences, in an article posted on a university website.

"In addition to that, all procedures are approved by Institutional Animal Care and Use Committees. It is essential to use these animals in vaccine studies before trials can be done in human subjects."

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Conspiracy Theorists Believe Jeffrey Epstein, Who Died in 2019, Is Alive at His New Mexico Ranch. He Is Not. – Inside Edition

Convicted sex offender and multimillionaire financier Jeffrey Epstein died by suicide in his Manhattan jail cell more than a year ago, but according to conspiracy theorists he is alive and well and at his ranch in New Mexico, the New York Post reported.

The YouTube channel ABQ Raw told viewers that Epstein could be seen in a red truck, near the fence line of the property of his ranch. The driver of the truck is said to have had a striking resemblance to the recently deceased Jeffrey Epstein. The poster says Epstein must have rode off into the sunset after faking his death, the New York Post reported.

Epstein, 66, did not fake his own death, but instead took his own life after beingcharged in July 2019 with the sexual trafficking of girls as young as 14.

Epstein conned people about his wealth, his financial stature and personal accomplishments, and deceived people in his pursuit to form valuable relationships with political and global leaders, scientists and other titans ofindustry, officials said. Epstein also hada fascination with transhumanism, the science of improving the human population through genetic engineering and artificial intelligence, The New York Times reported. Before hisdeath, Epstein hoped to seed the human race with his DNA by impregnating womenat his New Mexico ranch, the Times reported.

On Aug. 10, 2019, Epstein was found unresponsive in his cell in the Special Housing Unit of Manhattan Correctional Center in Lower Manhattan around 6:30 a.m. Jail staff tried to revive him, and then called for an ambulance.

He was taken to New York Downtown Hospital around 7:30 a.m. anddeclared dead a short time later. The city Medical Examiners office took Epsteins body from the hospital to the city morgue at Bellevue Medical Center Saturday afternoon to determine the cause of death, the Post reported.


Jeffrey Epstein's First-Known Accuser Says He Posed as Victoria's Secret Recruiter

Ghislaine Maxwell, Jeffrey Epstein Confidante and British Socialite, Arrested on Sex Charges

New York Coroner Rejects Claim That Jeffrey Epstein's Death Was 'Homicide'

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Conspiracy Theorists Believe Jeffrey Epstein, Who Died in 2019, Is Alive at His New Mexico Ranch. He Is Not. - Inside Edition

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High-tech medical and dental innovation garner the headlines but the most impactful practices are mostly lower tech and prevention-focused – Genetic…

However, in both medicine and dentistry, there is an important role as well for ingenious, low-tech, less expensive approaches to improved health and increased longevity.

The FDA last year approved a high-tech gene therapy drug, Zolgensma, for a rare childhood genetic disease, spinal muscular atrophy, that costs justover $2 millionfor the single dose of the treatment.The illness, which is caused by a defect in a gene calledSMN1, affects about 400 babies in the United States annually and kills those with the most common form of the disease in the first few years of life.The new treatment uses non-pathogenic, genetically engineered viruses to deliver healthy copies of theSMN1gene to patients cells so they can synthesize a protein needed to develop normal muscle neurons.

Another remarkable genetic engineering feat wasreportedin the journalNaturein 2017.An experimental gene therapy procedureused to transform and grow sheets of healthy skin saved the life of a 7-year-old boywho suffered from a genetic disease,junctional epidermolysis bullosa, that had blistered and destroyed most of his skin.He was on the verge of death, but two years after the treatment with genetically engineered cells produced by a multi-national team, he had healthy skin and was leading a normal life.

Those high-tech interventions are spectacular, but there are many simpler and cheaper yet tremendously important innovations for the diagnosis and prevention of illness.Among the most cost-effective are checklists for personnel in operating rooms and ICUs. According to a Norwegian research group, Safety checklists appear to be effective tools for improving patient safety in various clinical settings by strengthening compliance with guidelines, improving human factors, reducing the incidence of adverse events, and decreasing mortality and morbidity.

Sometimes, a simple tool or device is important to clinical diagnosis. One example is the hand-held direct ophthalmoscope, which allows a medical practitioner to look intotheback oftheeye to ascertain the health oftheretina, optic nerve, vasculature, and vitreous humor (the liquid inside the eyeball). Invented in 1851, it costs less than $200.

Another example is the way a singleblood testcan ascertain that a patient in the emergency room is not having a heart attack and so can forego the inconvenience and expense of additional invasive tests or unnecessary hospitalization.The highly sensitive blood test measures levels of cardiac troponin, a protein involved in muscle contraction; if the level is undetectable that is, below the limit of detection of the test there is a greater than 99% likelihood that the patient isnotexperiencing a heart attack and is at very low risk of other cardiac adverse events for at least 30 days.

That innovative approach is advantageous to patients and helps to reduce the frequency of hospitalizations and, therefore, healthcare costs.

Falls are both a cause and effect of declining health in the elderly.They are the leading cause of injury-related visits to emergency rooms and the primary cause of accidental deaths in Americans over the age of 65.To measure the potential benefits of a low-tech approach to preventing injuries from them, a research group in New Zealandcompared rates of falling and injuriesfrom falls on low-impact flooring (LIF) compared with standard vinyl flooring on an older persons health ward.Falls were prospectively monitored with written reports of all incidents, noting the location and consequences of each fall.The frequency of falls and injuries on LIF and those occurring on standard vinyl flooring (controls) were compared.

The investigators found that over the 31months of the study, there were 278 falls (among 178 persons who fell).The rate of falls was indistinguishable in the two groups, but fall-related injuries were significantly less frequent when they occurred on LIFs (22% of falls versus 34% of falls on control flooring).And many of the injuries that were averted were serious: Fractures occurred in 0.7% of falls in the LIF cohort versus 2.3% in the control cohort.

Thus, the New Zealand study provides a compelling rationale for adding low-impact flooring to housing for seniors (along withother modifications).

Dentistry has also benefited from costly high-tech innovations such asdental implants, but low-tech prophylaxis can also provide much needed benefits for dental health.

Tooth decay remains one of the majorpublic health concerns for both developing and developed countriesaccording to the World Health Organization.It is one of the most commonchronic problemsin the United States, where most adults will have at least one cavity in their lifetime. Decay causes inflammation in surrounding gum tissue, abscesses, and eventually, tooth loss. In addition to taking a significant toll on quality of life, decay and periodontitis has been linked to an increased risk of cardiovascular events, systemic infections such as endocarditis, and complications in pregnancy.

There have been significant advances in replacing and restoring teeth that need treatment due to dental caries, and these are often costly. However, preventing the problem in the first place is optimal, and an important advance was the introduction of water fluoridation, a simple low-tech intervention withproven efficacy, in 1945. The concept is that fluoride, a negatively charged ion, binds to calcium and phosphate on tooth surfaces to prevent the bacteria that cause cavities from even entering the tooth, thus protecting the teeth from dental decay and in some cases even reversing early decay. In a systematic review published in 2016, researchersfoundthat fluoridating drinking water in communities decreased overall decay and, thereby, the cost of more aggressive and costly dental interventions.

The Centers for Disease Control estimates that there are approximately100 million Americanswho are still without access to fluoridated water. Introducing this low-tech, high-impact measure more widely would substantially decrease the need for many dental procedures.

The high-tech miracles will continue to garner headlines, but to advance public health, simpler and relatively inexpensive innovations are also essential. That has policy implications: We need to put research dollars not only into potential big-ticket, high-tech blockbusters but also into ingenious, low-tech innovation.

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High-tech medical and dental innovation garner the headlines but the most impactful practices are mostly lower tech and prevention-focused - Genetic...

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SAB Biotherapeutics Awarded $57.5M from BARDA and US Department of Defense for Manufacturing of SAB-185 for the Treatment of COVID-19 | Antibodies |…

DetailsCategory: AntibodiesPublished on Tuesday, 01 December 2020 10:26Hits: 181

SIOUX FALLS, SD, USA I November 30, 2020 I SAB Biotherapeutics (SAB), a clinical stage biopharmaceutical company developing a novel immunotherapy platform to produce specifically targeted, high-potency, fully human polyclonal antibodies without the need for human serum, today announced that, as part of Operation Warp Speed, the Biomedical Advanced Research and Development Authority (BARDA), part of the Office of the Assistant Secretary for Preparedness and Response at the U.S. Department of Health and Human Services, and the Department of Defense Joint Program Executive Office for Chemical, Biological, Radiological and Nuclear Defense (JPEO-CBRND) have awarded SAB $57.5 million in expanded scope for its DiversitAb Rapid Response Antibody Program contract for the manufacturing of SAB-185, the companys clinical stage therapeutic candidate for COVID-19.

"We are pleased to be awarded this additional contract scope, which we believe is a reflection of the compelling science that supports SAB-185s potential in COVID-19, as well as the urgent need for treatment options amidst the global pandemic. Previous data has indicated that this human polyclonal antibody therapeutic has potent neutralizing activity against SARS-CoV-2, potentially driving more available doses, giving us the confidence to continue to progress our clinical development programs for SAB-185, said Eddie J. Sullivan, PhD, co-founder, president and CEO of SAB Biotherapeutics. This manufacturing agreement with BARDA and the Department of Defense supports our vision of bringing a novel, first-of-its-kind human polyclonal antibody therapeutic candidate for COVID-19 to patients, and I am proud of the work by our team and appreciate the continued support from BARDA and JPEO as we continue to rapidly advance SAB-185.

SAB-185 is currently being tested as a COVID-19 therapeutic in an ongoing Phase 1 trial in healthy volunteers and an ongoing Phase Ib trial in patients with mild or moderate COVID-19. SAB has leveraged its expertise to develop scalable manufacturing capabilities to support clinical activities, and continues to increase capacities in working with contract manufacturing organizations.

About SAB-185

SAB-185 is a fully-human, specifically-targeted and broadly neutralizing polyclonal antibody therapeutic candidate for COVID-19. The therapeutic was developed from SABs novel proprietary DiversitAb Rapid Response Antibody Program. SAB filed the Investigational New Drug (IND) application and produced the initial clinical doses in just 98 days from program initiation. The novel therapeutic has shown neutralization of both the Munich and Washington strains of mutated virus in preclinical studies. Preclinical data has also demonstrated SAB-185 to be significantly more potent than human-derived convalescent plasma.

About SAB Biotherapeutics, Inc.

SAB Biotherapeutics, Inc. (SAB) is a clinical-stage, biopharmaceutical company advancing a new class of immunotherapies leveraging fully human polyclonal antibodies. Utilizing some of the most complex genetic engineering and antibody science in the world, SAB has developed the only platform that can rapidly produce natural, specifically-targeted, high-potency, human polyclonal immunotherapies at commercial scale. SAB-185, a fully-human polyclonal antibody therapeutic candidate for COVID-19, is being developed with initial funding supported by the Biomedical Advanced Research Development Authority (BARDA), part of the Assistant Secretary for Preparedness and Response (ASPR) at the U.S. Department of Health and Human Services and the Department of Defense (DoD) Joint Program Executive Office for Chemical, Biological, Radiological, and Nuclear Defense (JPEO-CBRND) Joint Project Lead for Enabling Biotechnologies (JPL-EB). In addition to COVID-19, the companys pipeline also includes programs in Type 1 diabetes, organ transplant and influenza. For more information visit: or follow @SABBantibody on Twitter.

SOURCE: SAB Biotherapeutics

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Dlgap2: The Gene Associated With Memory Loss – Psychiatric Times

A new study associates a gene that facilitates neuron communication in the nervous system with memory loss.

A recent study1 led by The Jackson Laboratory and University of Maine has discovered that the gene Dlgap2 is associated with Alzheimer disease, dementia, and cognitive decline. Researchers found post-mortem human brain tissues of individuals experiencing poorer cognitive health and faster cognitive decline had low levels of Dlgap2.

The reason why this is so important is because a lot of research around cognitive aging and Alzheimers has been hyper-focused on well-known risk genes like APOE and brain pathologies, Catherine Kaczorowski, associate professor and Evnin family chair in Alzheimer disease research at The Jackson Laboratory (JAX), and adjunct professor with the University of Maine Graduate School of Biomedical Science and Engineering (GSBSE), said to the press. We wanted to give ourselves the option of looking at new things people keep ignoring because they've never heard about a gene before.2

Located in the synapses of neurons, Dlgap2 anchors critical receptors for signals between learning and memory neurons. The research team examined the memory and brain tissue from a large group of genetically diverse mice, relying on diversity outbred mice. The population came from 8 parents created by JAX, as they thought a diversified group would better reflect genetic diversity in humans. About 437 mice, eacheither 6, 12, or 18 months old were used.

Its great because you can harness the best parts of a mouse study and human society, Andrew Ouellette, a PhD student at JAX and a GSBSE NIH T32 predoctoral awardee, said to the press. Historically, research has been done with inbred mice with similar genetic makeups; same, similar genetic models. But clinically, humans don't work like that because they're not genetically identical.2

Quantitative trait loci mapping was performed on the mouse population. Study of entire genome sequences allowed for identification of the genes responsible for varying cognitive function and where they occurred. Researchers pinpointed the connection between Dlgap2 and memory decline in mice, and were then able to evaluate its significance to humans.

They found Dlgap2 is associated with the degree of memory loss in mice and risk for Alzheimer dementia in humans. Further research will be needed to determine how the gene influences dementia and brain functioning.


1. Ouellette AR, Neuner SM, Dumitrescu L, Hadad N, et al. Cross-species analyses identify Dlgap2 as a regulator of age-related cognitive decline and alzheimers dementia. Cell Reports. 2020;32(9):108091.

2. University of Maine. New connection between Alzheimer's dementia and Dlgap2. News release. ScienceDaily. November 23, 2020.

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