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Category Archives: Human Genetic Engineering

Does Autism Hold the Key to What Makes Humans Special? – The New York Times

Heres how the mechanism works: Humans alone observe the world and ask questions that demand why, how and what. They answer their questions by looking for if-and-then patterns, such as, if I boil an egg for eight minutes, then the yolk will be hard, and if I boil an egg for four minutes, then the yolk will be soft. They use those patterns to build theories, which they then repeatedly test, looking always for systems to further employ and exploit.

Grand theories aside, Baron-Cohen is at his most striking when he writes about people with autism, like Jonah, who was slow to talk but who taught himself to read. When Jonah eventually learned to speak, he used language less as a tool for communication than as a system for categorizing the world around him. As a young child, he was endlessly fascinated by how things worked, and he spent hours experimenting, like flipping a light switch on and off to test and retest its effect. At school he showed great brilliance in his observations about the natural world, he was a born pattern seeker, but at the same time he was taunted by other children for being so different. In group reading time, which he hated, he would shut his eyes and put his fingers in his ears. Jonahs weekend hobby as a young man was helping fishermen locate shoals by being able to read the signs from surface waves. Yet despite his incredible talents, Jonah was lonely and frustrated because he couldnt find a job that would allow him to live an independent life. Baron-Cohen argues with feeling and conviction that society must do a better job of making room for people like Jonah, and that it will benefit enormously when it does.

Mostly, though, The Pattern Seekers is about the idea of using autism as a key to unlock the mystery of human cognition, and on this front, its less convincing. Sometimes its simply because the books framing is misleading. Baron-Cohen takes great care to set up the idea that all humans possess a Systemizing Mechanism, that some people are hyper-systemizers, and that a comparatively high number of those hyper-systemizers are autistic. But the subtitle of the book is not how systemizing drives human invention, its how autism drives human invention. At the same time, he cautions against speculation that people, living or dead, might be autistic. The term should be reserved only for diagnosis when people are struggling to function, he explains.

In addition, Baron-Cohen divides humans into five brain types, grouping people who are more or less likely to systemize or empathize. He believes that humans also uniquely possess an Empathy Circuit. But he establishes his five groups by conducting large surveys about individual tendencies and traits, so they are not brain types at all. They are, at best, mind types.

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Does Autism Hold the Key to What Makes Humans Special? - The New York Times

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Maze Therapeutics and Alloy Therapeutics Form Broadwing Bio to Develop Antibody Therapies for Genetically Validated Targets in Ophthalmic Diseases -…

SOUTH SAN FRANCISCO, Calif. & LEXINGTON, Mass.--(BUSINESS WIRE)--Maze Therapeutics, a company focused on translating genetic insights into new medicines, and Alloy Therapeutics, a company developing platforms and services to enable drug discovery, today announced the formation of Broadwing Bio to develop targeted antibody therapies for the treatment of ophthalmic diseases. Broadwing Bio will advance programs directed to genetically validated ophthalmology targets identified using Mazes human genetics and functional genomics platform, the COMPASS platform. Alloy Discovery Services will generate therapeutic candidates using Alloys broad suite of antibody discovery technologies, including the ATX-Gx mouse platform.

Under the terms of the agreement, Maze and Alloy will fund Broadwing Bio to rapidly advance its programs through preclinical and clinical development with the opportunity for independent financing and partnering. Maze and Alloy will retain certain rights to participate in the development and commercialization of products originating from Broadwing Bio. The company will be led by Andrew Peterson, Ph.D., founder and chief executive officer of Broadwing Bio.

We are very excited to partner with Alloy on the formation of Broadwing Bio, with a mission to advance therapeutics for ophthalmology indications, said Jason Coloma, Ph.D., chief executive officer of Maze. Maze is advancing a pipeline of programs based on genetic insights of disease, without restrictions on modality or therapeutic area. This joint venture will allow us to pursue compelling Maze-identified targets through a dedicated organization with the experience and focus to develop highly differentiated therapies addressing unmet needs in ophthalmology, while retaining significant financial participation and product rights.

Broadwing Bio is a great example of the high impact partnerships Alloy Discovery Services will conduct on a very select basis, where we can invest heavily in the success of the partnership, said Errik Anderson, chief executive officer and founder of Alloy Therapeutics. We are honored to be working with an incredible scientist-entrepreneur like Andy, in partnership with Mazes team, to advance these exciting drug targets designated by Maze.

Broadwing Bio has established a team of experienced leaders and scientific advisors, including

There are a number of ophthalmic diseases for which effective therapeutic options are limited, but recent genetic insights provide avenues to change this situation, said Dr. Peterson. Broadwing Bio has the very unique opportunity to bring together the capabilities of two exceptional companies in order to develop novel treatments targeted at these diseases. Im thrilled to join the company as CEO and look forward to building the Broadwing Bio team, while leveraging Maze and Alloys insights and experience in drug discovery in order to bring medicines to patients in need.

About Maze Therapeutics

Maze Therapeutics is a biopharmaceutical company developing a broad portfolio of therapeutic candidates for a number of genetically defined diseases. Maze is focused on translating genetic insights into new medicines by utilizing an approach that combines the analysis of large-scale human genetics data, cutting-edge functional genomics and an array of drug discovery approaches. The Maze COMPASS platform reveals modifier genes that confer protection and provides deeper understanding of the target biology and how these targets can be best targeted with drug therapies. Maze was launched in 2019 by Third Rock Ventures, with funding from ARCH Venture Partners, GV, Foresite Capital, Casdin Capital, Alexandria Venture Investments, City Hill and other undisclosed investors. Maze is based in South San Francisco. For more information please visit mazetx.com.

About Alloy Therapeutics

Alloy Therapeutics is a biotechnology company dedicated to empowering scientists in the relentless pursuit of making better medicines for all. To this end, Alloy seeks to democratize access to foundational drug discovery platforms and services to scientists worldwide. Alloys first platform, the ATX-Gx mouse platform, is a suite of transgenic mice designed for best-in-class in vivo discovery of fully human monoclonal antibodies. Alloys partners include academic scientists, small and medium biotech, and Fortune 50 biopharma. Founded in 2018 and privately funded by visionary investors, Alloy Therapeutics is headquartered in Boston, Massachusetts with European labs in Cambridge, UK. As a reflection of our irrational commitment to the scientific community, 100% of our revenue from platforms and services is reinvested in innovation and supporting access to innovation. To join the revolution, visit alloytx.com or schedule a 15-minute chat with our Founder and CEO at alloytx.com/ceo.

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Maze Therapeutics and Alloy Therapeutics Form Broadwing Bio to Develop Antibody Therapies for Genetically Validated Targets in Ophthalmic Diseases -...

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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 – KSL.com

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

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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.

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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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