Breakthrough gene therapy alleviates Dravet syndrome symptoms in mice without side effects
In a groundbreaking advancement for families grappling with the challenges of Dravet syndrome, a rare and life-altering form ...
EurekAlert!3d
Breakthrough Dravet syndrome gene therapy in mice brings new hope to families
In a groundbreaking advancement for families grappling with the challenges of Dravet syndrome, a rare and life-altering form ...
Gene therapy loses luster as investors eye quicker returns from weight-loss drugs
Gene therapy, with its offer of a possible cure for rare diseases like sickle cell, is losing early investors to ...
The American Journal of Managed Care3d
Bustling Gene Therapy Pipeline for Neuromuscular Diseases Brings Thorny Questions to the Clinic
The rapid development of gene therapy options for treating neuromuscular diseases has created new therapeutic options but ...
STAT1d
Gene therapies and CRISPR drugs slowed by ‘irrational pessimism,’ say scientists forging new paths
Gene therapy pioneer Jim Wilson and researcher Kiran Musunuru are using contrasting approaches to overcome “irrational ...
GEN - Genetic Engineering and Biotechnology News2d
Gene Therapy Tested in Mice Offers New Hope for People with Dravet Syndrome
By replacing the defective gene associated with Dravet syndrome in mice, scientists successfully alleviated symptoms without ...
The American Journal of Managed Care4d
Shaping Gene Therapy Approaches in Neuromuscular Disease
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 ...
BioPharma Dive1d
Novartis builds case for new SMA gene therapy
To Novartis, fresh late-stage data support the idea that its intrathecal drug, which has the same active ingredient as ...
University of California1d
Breathing new life: Working toward an inhalable gene therapy for cystic fibrosis
Although cystic fibrosis is a single gene mutation, there are more than 1,000 different ways the CFTR gene can mutate in ...