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

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 ...

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

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

After scoring tax abatements for a $100 million investment in The Woodlands last year, the company appears to be close to beginning the project.

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

Immuno-oncology and cell-based treatments, which harness the body's own immune system to combat illnesses like cancer, have ...

The study aimed to develop and evaluate a gene replacement therapy for DEE52 using an adenovirus-associated virus (AAV) vector encoding circular DNA (cDNA) for the β1 subunit, termed AAV-Navβ1.

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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 ...

The future of medicine is being shaped by a confluence of factors at play. If there are worries galore around drug resistant ...