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

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

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 ...

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 ...

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

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

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 ...

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 ...

(Yicai) Jan. 28 -- Chinese medical researchers have made a breakthrough in gene therapy that overcomes the limitations of ...

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