Researchers described a promising new approach for using gene therapy to treat sickle cell disease in the journal Human Gene ...

Researchers at Children's Hospital of Philadelphia (CHOP) announced encouraging results from the first ever gene therapy ...

Gene therapies have a lot of potential to improve health outcomes for patients with life-threatening diseases but face a ...

CHICAGO -- Gene therapy for Danon disease, a rare inherited cause of hypertrophic cardiomyopathy, appeared to improve or ...

Researchers have developed a dual-gene therapy approach to treat Usher syndrome type 1F, a rare condition causing deafness and progressive blindness.

Cyclosporin H boosts a new lentivirus-delivered gene therapy with enhanced anti-sickling properties in blood stem cells from ...

A new type of therapy that ‘edits’ a gene in patients with a rare heart condition has been shown to be safe and effective, ...

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

A gene is a specific sequence of DNA that carries the instructions for making a protein. In non-integrating gene therapy, a piece of DNA with a correct copy of the CFTR gene is delivered to an ...

Clinical Trial Success at Cincinnati Children's Supports Recent US FDA Approval for First Therapy to Directly Replace a Gene in the Brain CINCINNATI, Nov. 15, 2024 /PRNewswire/ -- Sriansh Ojha ...

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

Delivering very large genes to cells can pose a serious challenge, so scientists developed a new method. | Genetics And ...