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 ...

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 ...

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

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 ...

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 ...

The disease leads to the progressive growth of fluid-filled cysts in the kidneys, often resulting in kidney failure and other ...

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; ...