In 1962, a pediatric neurology resident at Columbia's Neurological Institute and his colleagues in the College of Physicians ...
The Boston biotech founded by Harvard professor David Sinclair is testing whether cellular reprogramming can turn back the ...
A top US regulator plans to unveil a faster approach to approving custom gene-editing treatments, a move designed to unleash a wave of industry investment that will yield cures for patients with rare ...
It’s now possible to treat inherited blood diseases, such as sickle cell disease, with gene editing. Blood stem cells are extracted from the patient, modified, and infused back into their bone ...
Long-term follow-up shows 95% success rate, no serious complications in largest ADA-SCID gene therapy study to date ...
The U.S. Food and Drug Administration (FDA) on Monday extended its review timeline for REGENXBIO Inc.’s (NASDAQ:RGNX) Biologics License Application (BLA) for clemidsogene lanparvovec (RGX-121) for ...
Some genetic disorders—such as cystic fibrosis, hemophilia and Tay Sachs disease—involve many mutations in a person's genome, ...
Genprex, Inc. ("Genprex" or the "Company") (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing ...
Preliminary results from a small trial offer the clearest evidence yet that the brain disease’s progression can be slowed. A one-time gene therapy can markedly slow the progression of Huntington’s ...
In a study of human immune cells infected with HIV, the virus that causes AIDS, scientists at Johns Hopkins Medicine say a molecule within HIV itself can be manipulated and amplified to force the ...
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