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 ...

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

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

The FDA has placed clinical holds on a pair of investigational gene therapies developed to address two rare neurodevelopmental disorders in children, according to the manufacturer. In a press release, ...

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

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

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

The Food and Drug Administration paused trials for two experimental gene therapies from Regenxbio after one child developed a ...

Gene-editing tools like CRISPR have unlocked new treatments for previously uncurable diseases. Now, researchers at the ...

The special properties of methylcellulose foam could make it a vehicle for bedside genetic engineering, according to a proof-of-principle study from bioengineers at Fred Hutch Cancer Center. In the ...

Studies of Hunter and Hurler syndromes on hold ...