Gene therapy represents one of medicine’s most ambitious attempts to treat disease at its root cause by altering the genetic code itself. The approach works by recovering the functions of critical ...

When gene therapy was first proposed in the 1970s, the idea seemed straightforward but revolutionary: replacing defective genes with functional ones to cure a disease. But over the decades, this ...

Onasemnogene abeparvovec gene therapy post-nusinersen or risdiplam showed meaningful motor improvements in children with SMA, with a manageable safety profile. The study cohort included older, heavier ...

Results from the largest cohort of children who received a gene therapy for a rare immunodeficiency condition have shown the long-term safety and efficacy of the curative treatment, in a study led by ...

SEATTLE — Some of our most challenging health conditions result from genetic disorders like sickle cell disease and certain cancers. Gene therapy is revolutionizing how they're treated, and the Fred ...

DMD gene therapy faces challenges in safety, efficacy, and long-term outcomes, despite FDA approval of delandistrogene moxeparvovec. Future strategies focus on novel AAV capsids, dual/triple vector ...

Gazi University in Ankara has officially launched the production process for Türkiye's first local and national gene therapy research product. The project, which targets rare genetic diseases, is ...

After a series of deaths in patients taking Sarepta Therapeutics’ gene therapies, doubt has crept into investor sentiments ...

Please provide your email address to receive an email when new articles are posted on . An infant with a rare urea cycle disorder became the first patient to receive a personalized gene-editing ...

Forbes contributors publish independent expert analyses and insights. A patient in a late-stage gene editing trial for a rare heart condition died from fatal liver complications after receiving an ...