StitchR, a new gene therapy technique, delivers large genes in two parts to treat muscular dystrophies by restoring critical proteins in animal models.

Harvard Medical School researchers have taken another decisive step in their efforts to develop a gene therapy for people ...

a universal gene therapy targeting GATA1. This new method could transform DBA treatment by overcoming the genetic diversity ...

Nov 13 (Reuters) - The U.S. Food and Drug Administration approved PTC Therapeutics' (PTCT.O), opens new tab gene therapy to ...

Sriansh Ojha was fully into his "terrible twos" on an April afternoon at Cincinnati Children's as he squealed and chased his ...

Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is ...

An RNA-editing gene therapy has been developed that switches off the key driver of common eye conditions affecting diabetics ...

The FDA issued its first stamp of approval for a cell or gene therapy back in 2017 to Novartis' Kymriah. | The gene therapy ...

University of Wisconsin–Madison researchers targeting a group of hereditary neurodegenerative diseases have found success ...

Kebilidi is the first approved gene therapy that can be directly administered to the brain. Its OK secures a priority review ...

Gene therapy, the idea of fixing faulty genes ... Scientists Discover New Code Governing Gene Activity Aug. 20, 2024 — A newly discovered code within DNA -- coined 'spatial grammar' -- holds a ...

UW–Madison researchers used gene therapy to prevent hereditary spastic paraplegia (HSP) in a rat model, introducing a healthy ...