Intellia Therapeutics NTLA announced that the FDA has granted the Regenerative Medicine Advanced Therapy (RMAT) designation ...

Childhood-onset striatonigral degeneration is a rare genetic disorder that robs children of the ability to walk and speak by ...

Sebastien Beauzile, 21, is the first New Yorker to have received the breakthrough Lyfgenia treatment, according to the New ...

R75W mutation in the gap junction β2 (GJB2) gene causes severe fragmentation of gap junction plaques, connecting adjacent ...

The field of gene editing recently got a major boost from Massachusetts-based Beam Therapeutics, and the company’s Durham ...

Congenital hearing loss refers to impaired auditory function that occurs due to genetic causes. GJB2 is the gene responsible ...

Aug. 29, 2024 — For decades, scientists have dreamt of a future where genetic diseases, such as the blood clotting disorder hemophilia, could be a thing of the past. Gene therapy, the idea of ...

An R75W mutation in the gap junction β2 (GJB2) gene causes severe fragmentation of gap junction plaques, connecting adjacent ...

In the time since the Novartis gene therapy Zolgensma was approved for babies with spinal muscular atrophy, other treatments ...

A pair of privately-held developers of editing-based therapies have some successes to show for their recent efforts to ...

For the first time, scientists say, they have evidence that using a biologic drug to remove sticky beta amyloid plaques from ...