In his CEO Commentary, Frederic Revah, Ph.D., underscores the dedication, expertise and successes of Genethon's 240 scientists and professional staff as they pursue new gene therapy treatments for ...

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

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

A University of California San Diego-led team has discovered that restoring a key cardiac protein called connexin‑43 in a ...

Experimental gene therapies like AMT‑130 lower toxic huntingtin protein and significantly slow Huntington’s disease, offering ...

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

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 ...

A new study has brought scientists one step further in the direction of developing a cure for a brutal group of rare brain disorders known as SYNGAP1-related disorders, or SRDs. Researchers were able ...

Under the agreement, participating states will receive “discounts and rebates” from the drugmakers if the treatments don’t ...

A new CRISPR-based one-off procedure that lowers "bad" cholesterol has been approved to enter Phase I human trial. If ...

For the first time, the tools to rewrite human biology are moving from speculative fiction into regulated clinics and ...

Researchers have discovered that eight different psychiatric conditions share a common genetic basis. A study published in ...