2024 — Using state-of-the-art technology, researchers have identified several specific steps needed for CRISPR to become active and perform its gene editing ... Single-Dose Gene Therapy Is ...
In a groundbreaking advancement for families grappling with the challenges of Dravet syndrome, a rare and life-altering form ...
Gene therapy pioneer Jim Wilson and researcher Kiran Musunuru are using contrasting approaches to overcome “irrational ...
Scientists at the Allen Institute have made a major breakthrough in research for people living with Dravet Syndrome, which is ...
By replacing the defective gene associated with Dravet syndrome in mice, scientists successfully alleviated symptoms without ...
Although cystic fibrosis is a single gene mutation, there are more than 1,000 different ways the CFTR gene can mutate in ...
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 ...
Shares of Sarepta Therapeutics have struggled for the past year due to concerns about the commercial potential of its gene therapy and broader pipeline. Safety hadn’t been a major worry for ...
In a groundbreaking advancement for families grappling with the challenges of Dravet syndrome, a rare and life-altering form ...
A pair of privately-held developers of editing-based therapies have some successes to show for their recent efforts to ...
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Breakthrough gene therapy in Seattle offers hope for children with epilepsyThe research, taking place at the Allen Institute for Brain Science, offers new hope to families fighting against Dravet Syndrome, a severe form of epilepsy that devastates childhood development.
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