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

ACM impedes the heart from pumping blood to the rest of the body, and is a leading cause of sudden cardiac death in young ...

A gene therapy candidate extended survival in a mouse model of SMARD1, supporting its testing in a clinical trial for this ...

As newborn screening and rapid DNA sequencing become routine, we are poised to catch and treat inherited diseases at their earliest stages. Today, we can intervene in the first days or weeks of life.

Universal gene‑edited CAR‑T therapies turn donor T cells into off‑the‑shelf cancer treatments, cutting delays and costs while ...

Gene therapy is becoming a powerful way to treat challenging diseases that don’t respond to traditional treatments, and researchers now report the first success in modifying genes to slow Huntington’s ...

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

Up to three in every 1,000 newborns is born with hearing loss in one or both ears. While cochlear implants have long been a life-changing option, they involve surgery and can't fully replicate the ...

Youth with congenital deafness treated with gene therapy showed better progress on certain hearing measures than counterparts treated with cochlear implantation in a cohort study from China. Gene ...