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

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 ...

With a shared purpose, we can realize the true promise of CRISPR and improve healthcare, providing hope to more patients with ...

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

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

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 ...

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

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; ...