Gene therapy, with its offer of a possible cure for rare diseases like sickle cell, is losing early investors to ...
Dec. 10, 2024 — In a single IV injection, a gene therapy targeting cBIN1 can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the ...
Gene therapy pioneer Jim Wilson and researcher Kiran Musunuru are using contrasting approaches to overcome “irrational ...
Advances in the gene-editing technology known as CRISPR-Cas9 over the past 15 years have yielded important new insights into the roles that specific genes play in many diseases.
A pair of privately-held developers of editing-based therapies have some successes to show for their recent efforts to ...
Although cystic fibrosis is a single gene mutation, there are more than 1,000 different ways the CFTR gene can mutate in ...
To Novartis, fresh late-stage data support the idea that its intrathecal drug, which has the same active ingredient as ...
In a groundbreaking advancement for families grappling with the challenges of Dravet syndrome, a rare and life-altering form ...
In a groundbreaking advancement for families grappling with the challenges of Dravet syndrome, a rare and life-altering form ...
Adeno-associated virus (AAV)–mediated gene therapy was one of the focal points of this year's Muscular Dystrophy Association conference. Here, Barry Byrne, MD, PhD, University of Florida, speaks to ...
Gene Hackman's cause of death was heart disease, wife died of hantavirus days earlier, officials say
New Mexico officials have announced the cause of death for actor Gene Hackman and his wife Betsy Arakawa, a week after they were found dead at their home in Santa Fe. Hackman died of heart disease ...
SANTA FE, N.M. — A towering figure in cinema for decades, retired actor Gene Hackman spent his final years in seclusion with his wife, living with heart disease and Alzheimer's, authorities said ...
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