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

Sebastien Beauzile, 21, became the first person in New York state to be made symptom-free of sickle cell disease after ...

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

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 ...

NY resident Sebastien Beauzile was treated with a pioneering new gene therapy approach called Lyfgenia, curing his sickle ...

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

Typically, as a person gets older, the gapping of the end of the telomere chromosomes gets shorter, allowing DNA strands to separate ... Elsewhere in the field of gene therapy for rare disorders, An ...

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

Danon disease is a rare but serious cardiomyopathy that drastically reduces life expectancy. Male patients infrequently live ...

With MeiraGTx eyeing up how to take the drug through phase 3 and onto commercialization, Hologen has stepped in. The ...

New findings show that the mitochondria powering our cells also control the ability of a DNA repair protein to suppress the senescence-associated secretory phenotype (SASP), which causes zombie-like ...

or slicing DNA to silence genes. Dr. Musunuru is a co-founder and equity holder of Verve Therapeutics, a gene therapy company, and receives funding from Beam Therapeutics for research, but not for ...