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

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

While the existing gene therapy was approved for use in Europe in 2024, it is very expensive, which limits its accessibility. Moreover, this treatment modifies a globin repressor gene, which indeed ...

PHILADELPHIA, PA — As it prepares to test a first-of-its-kind gene therapy in patients, Nuevocor has named industry veteran Al Gianchetti as its new chief executive officer, a leadership move ...

Onasemnogene abeparvovec gene therapy post-nusinersen or risdiplam showed meaningful motor improvements in children with SMA, with a manageable safety profile. The study cohort included older, heavier ...

New one-dose treatment edits a gene in the liver, disabling the ANGPTL3 protein and significantly lowers cholesterol.

Until recently, even the most advanced gene therapies could only be given after a child was born—often racing against time to prevent irreversible damage. In the first part of this series, we explored ...

Opus Genetics is conducting a trial for OPGx-MERTK gene therapy in Abu Dhabi, targeting MERTK-related retinitis pigmentosa using AAV-based delivery. The trial is a collaboration with Cleveland Clinic ...

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

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 ...

Forbes contributors publish independent expert analyses and insights. A patient in a late-stage gene editing trial for a rare heart condition died from fatal liver complications after receiving an ...

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 ...