UC San Diego scientists have created a gene therapy that goes beyond masking Alzheimer’s symptoms—it may actually restore ...

A new gene therapy delivery device could let hospital pharmacies make personalized nanomedicines to order. This democratized ...

Pompe's disease is a lysosomal storage disorder caused by a genetic deficiency of GAA. The disease is associated with severe ...

Researchers have developed a gene therapy that significantly slowed motor function loss in preclinical models of amyotrophic ...

Gene therapy is a revolutionary field in modern medicine, offering transformative potential to treat and potentially cure a wide range of genetic and acquired diseases. By addressing the root ...

For patients with recessive dystrophic epidermolysis bullosa (RDEB), a one-time surgical application of prademagene ...

Genetically engineered skin grafts exhibited a long-term ability to heal large, chronic wounds in patients with recessive ...

Isaralgagene civaparvovec is a “potential best-in-class gene therapy for Fabry disease,” according to analysts at H.C.

Personalized gene editing saved a baby, but the tech’s future is uncertain. Costs, government regulation and lack of know-how could hinder wider use of the CRISPR tech ...

News broke yesterday that researchers in Philadelphia appear to have successfully treated a 6-month-old baby boy, called KJ, with a personalized CRISPR gene-editing therapy. The treatment corrects ...

Gene therapy fixes that problem by giving the patient’s blood-forming cells a new, normal hemoglobin gene. An estimated 100,000 people in the United States, most of them Black, have sickle cell ...

Genetics researcher Jonathan Rios talks about his research to test a gene therapy for a rare neurological disease in children called childhood-onset striatonigral degeneration, at Dallas Scottish ...