Delivery of therapeutic genes is essential for gene therapy. Adeno-associated viruses (AAVs) are a prime vector for carrying ...
Gene-editing tools like CRISPR have unlocked new treatments for previously uncurable diseases. Now, researchers at the ...
Gene therapy is a medical treatment that works by changing or fixing a person's genes to treat or prevent disease ...
The Food and Drug Administration paused trials for two experimental gene therapies from Regenxbio after one child developed a ...
Dr. Rebecca Ahrens-Nicklas and Dr. Kiran Musunru crafted a bespoke treatment that has successfully corrected the genetic defect.
Lilly and German biotech Seamless Therapeutics will develop recombinase-based treatments for hearing loss.
The disease leads to the progressive growth of fluid-filled cysts in the kidneys, often resulting in kidney failure and other ...
Two-year follow-up data demonstrate sustained benefit, including improvements in pain, fatigue and bone health, following single low dose of avigbagene parvec (FLT201) ...
Particle therapy, including proton and carbon ion radiotherapy, represents a major advancement in cancer treatment due to its ...
A five-year-old patient dosed with RGX-111 has developed a brain tumour four years after being dosed with the gene therapy.
Studies of Hunter and Hurler syndromes on hold ...