A research team has successfully demonstrated the world's first gene-editing treatment for Leber's hereditary optic ...

Gene therapy holds the promise of preventing and curing disease by manipulating gene expression within a patient's cells. However, to be effective, the new gene must make it into a cell's nucleus. The ...

Groundbreaking advancements in gene therapy at UTMB represent new possibilities for patients living with rare genetic ...

Life Biosciences is pushing cellular rejuvenation into the clinic with ER-100, an experimental gene ...

Gene therapy holds the promise of preventing and curing disease by manipulating gene expression within a patient's cells. However, to be effective, the new gene must make it into a cell's nucleus. The ...

Gene therapy is a medical treatment that works by changing or fixing a person's genes to treat or prevent disease ...

Delivery of therapeutic genes is essential for gene therapy. Adeno-associated viruses (AAVs) are a prime vector for carrying ...

Dr. Rebecca Ahrens-Nicklas and Dr. Kiran Musunru crafted a bespoke treatment that has successfully corrected the genetic ...

Sangamo Therapeutics recently reported detailed data from its completed registrational Phase 1/2 STAAR study of isaralgagene ...

University of California San Diego-led team has discovered that restoring a key cardiac protein called connexin‑43 in a mouse ...

A research team led by Professor Hyunji Lee of the Department of Convergence Medicine at Korea University College of Medicine (co-first authors: PhD candidates Sanghun Kim, Ji Eun Kim, and Sungjin Ju; ...

By Mariam Sunny Jan 26 (Reuters) - Sarepta Therapeutics' gene therapy for Duchenne muscular dystrophy slowed disease ...