The small study in patients with a rare disorder that causes liver and lung damage showed the potential for precisely ...

In the time since the Novartis gene therapy Zolgensma was approved for babies with spinal muscular atrophy, other treatments ...

Sarepta Therapeutics said on Tuesday a young man has died due to acute liver failure after treatment with its gene therapy for a rare muscular dystrophy.

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 therapy, the idea of fixing faulty genes ... Scientists Discover New Code Governing Gene Activity Aug. 20, 2024 — A newly discovered code within DNA -- coined 'spatial grammar' -- holds a ...

Shortly after this publication, Bogdanove struck up a collaboration with Daniel Voytas, a plant geneticist at the University ...

In a preclinical study, researchers at Children's Hospital of Philadelphia (CHOP) demonstrated a novel gene therapy with ...

Gene therapy involves the introduction of new genes into cells, to restore or add gene expression, for the purpose of treating disease. Most commonly a mutated gene is replaced with DNA encoding a ...

The new lentiviral vector-based gene therapy works by inserting a functioning copy of the CFTR gene in the DNA of the epithelial cells in a patient's airway. At present, some people with CF ...

The one-time therapy appears to correct the mutation that causes the disease, known as Alpha-1 antitrypsin deficiency (AATD), ...

While initial study results suggest Beam's technology can correct alpha-1 antitrypsin deficiency's genetic roots, shares fell ...

Giulio Cossu, MD, speaks to the lingering safety concerns related to ex vivo gene therapy in Duchenne muscular dystrophy (DMD) as long-term data are yet to be established.