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

Two dose-limiting toxicities have been recorded in uniQure’s trial evaluating an investigational AAV gene therapy designed to ...

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

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 ...

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 ...

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

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

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

AskBio Inc., an RTP-based gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, has received U.S. Food and Drug Administration (FDA) acceptance of its ...

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 ...

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