A "gene silencer" (technically known as small interfering RNA, or siRNA), locally delivered by nanoparticles embedded in an ...

Neurodegenerative diseases (NDDs) represent a profound challenge to global health, characterized by progressive neuronal dysfunction and loss, leading to ...

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

Scientists from UC Davis Center for Surgical Bioengineering, the MIND Institute and UC Berkeley’s Murthy Lab are developing ...

Lozano is a rare disease mom, neuroscience Ph.D. candidate at UC Davis, and board member for the PURA Syndrome Foundation. In May, a historic moment in science and medicine was captured in a single ...

Genethon, a leading laboratory in gene therapy for rare diseases, today announced that it has entered into an exclusive, worldwide licensing agreement with AskBio, a gene therapy company wholly owned ...

Fractyl Health is developing a shot that would program the body to make more of the GLP-1 hormone naturally, a risky bet that it can provide a longer-lasting benefit than blockbuster weight-loss drugs ...

The gene therapy market is surging toward $36.55 billion by 2032, powered by rising demand for curative treatments that target genetic diseases at their source([1]) . The FDA approved three ...

Live Science's health channel editor makes predictions about the medical breakthroughs and public health shifts to come in 2026. When you purchase through links on our site, we may earn an affiliate ...

Researchers have demonstrated the ability to deliver a fully functional copy of the CLN3 gene to stem cells of patients with juvenile NCL, an inherited neurodegenerative disease in which a mutation in ...

Hormone replacement therapy can be a way for women to prevent the inflammation that leads to disease. "There's some bone ...

Tenaya Therapeutics recently outlined its 2026 priorities, including updates on its TN-201 and TN-401 cardiac gene therapy programs and the resumption of patient enrollment in key Phase 1b/2 trials ...