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CRISPR gene editing therapy for HIV is heading into human testing after FDA clearance

BY ANNALEE ARMSTRONG FIERCE BIOTECH

A CRISPR-Cas9 gene editing technology that has shown promise in clearing HIV from mice is headed into human testing. Excision BioTherapeutics will usher the CRISPR-based therapy EBT-101 into clinical trials after the FDA cleared an investigational new drug application, according to the company’s press release. EBT-101 is under development as a potential virus-clearing treatment for patients with HIV—or, put in the company’s words, “a potential functional cure for chronic HIV.”

We don’t like to throw the word “cure” around here. But Excision thinks the therapy could replace standard-of-care retroviral therapy, which keeps HIV from replicating but does not remove it from the body. That means patients stay on the treatment, which can cause serious side effects and affect quality of life. Continue Reading →

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First genetically modified mosquitoes released in the United States

BY EMILY WALTZ
NATURE
After a decade of fighting for regulatory approval and public acceptance, a biotechnology firm has released genetically engineered mosquitoes into the open air in the United States for the first time. The experiment, launched this week in the Florida Keys — over the objections of some local critics — tests a method for suppressing populations of wild Aedes aegypti mosquitoes, which can carry diseases such as Zika, dengue, chikungunya and yellow fever. Continue Reading →

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Scientists win historic Nobel chemistry prize for ‘genetic scissors’

BY PAUL RINCON BBC SCIENCE

Two scientists have been awarded the 2020 Nobel Prize in Chemistry for developing the tools to edit DNA. Emmanuelle Charpentier and Jennifer Doudna are the first two women to share the prize, which honours their work on the technology of genome editing. Their discovery, known as Crispr-Cas9 “genetic scissors”, is a way of making specific and precise changes to the DNA contained in living cells. They will split the prize money of 10 million krona (£861,200; $1,110,400). Biological chemist Pernilla Wittung-Stafshede, commented: “The ability to cut DNA where you want has revolutionised the life sciences.” Continue Reading →

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Did coronavirus come from a lab?

BY MICHAEL MARSHALL
NEWSCIENTIST

Researchers led by Shan-Lu Liu at the Ohio State University say there is “no credible evidence” of genetic engineering. The virus’s genome has been sequenced, and if it had been altered, we would expect to see signs of inserted gene sequences. But we now know the points that differ from bat viruses are scattered in a fairly random way, just as they would be if the new virus had evolved naturally. Continue Reading →

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Quest to use CRISPR against disease gains ground

BY HEIDI LEDFORD
NATURE

The prospect of using the popular genome-editing tool CRISPR to treat a host of diseases in people is moving closer to reality.

Medical applications of CRISPR–Cas9 had a banner year in 2019. The first results trickled in from trials testing the tool in people, and more trials launched. In the coming years, researchers are looking ahead to more sophisticated applications of CRISPR genome editing that could lay the foundation for treating an array of diseases, from blood disorders to hereditary blindness. Continue Reading →

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There Still Aren’t Any Rules Preventing Rogue Scientists From Making Gene-Edited Babies

BY EMILY MULLIN
MEDIUM / ONE-ZERO
Around this time last November, Chinese scientist He Jiankui stunned the world when he revealed the birth of the first known gene-edited babies. Working in relative secrecy, he had used CRISPR to modify human embryos in the lab and then established pregnancies with those embryos. Twin girls with edited genomes were born as a result.
The scientific community’s condemnation of He was harsh and swift. He had edited the germline, making a heritable genetic change. There were safety questions about the effects on the twins, and he had not meant to fix a genetic defect or prevent disease. Instead, he tweaked a gene in an attempt to bestow an uncommon, protective genetic trait: resistance to HIV.
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