Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is a big problem: size.

Lisa Bancroft is among thousands of people who have been tested for the faulty BRCA gene since its discovery 30 years ago.

The Food and Drug Administration (FDA) has granted accelerated approval to Kebilidi™ (eladocagene exuparvovec-tneq) for the treatment of adult and pediatric patients with aromatic L-amino acid ...

University of Washington-led research, published in 2023, discovered microglia in the brains of people with Alzheimer's ...

Although gene therapies targeting specific genetic mutations in hematopoietic stem and progenitor cells (HSPCs) have shown ...

Researchers have identified new roles for a protein long known to protect against severe flu infection – among them, raising the minimum number of viral particles needed to cause sickness.

StitchR, a new gene therapy technique, delivers large genes in two parts to treat muscular dystrophies by restoring critical proteins in animal models.

In a phase 2 trial, a gene therapy reduced angioedema attacks, which could significantly change the quality of life for ...

Read updates from clinical trials and scientific research on Huntington’s disease from Day 3 of the 2024 Huntington Study ...

Recent research has revealed that alcohol use disorder and Alzheimer’s disease share significant patterns of gene ...

Enrollment and dosing complete in single ascending dose clinical trial of anti-tau antibody VY7523 for Alzheimer's; on track to generate top-line data H1 2025 - - Recent third-party clinical data esta ...

Highly effective treatments for HIV have existed since the mid-1990s. But while these treatments keep people healthy, we do not yet have a safe and scalable way to completely rid the body of the virus ...