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 ...

GENE202 is a single dose gene therapy for patients with rare metabolic disease, methylmalonic acidaemia.

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 ...

Ultragenyx now expects a 6-month review period for the therapy and a likely decision from the FDA regarding potential ...

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

Two-year follow-up data demonstrate sustained benefit, including improvements in pain, fatigue and bone health, following single low dose of avigbagene parvec (FLT201) ...

U.S. drugmaker Eli Lilly and Company signed an agreement worth up to $1.12 billion with Seamless Therapeutics, the ...

Studies of Hunter and Hurler syndromes on hold ...

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

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