The novel treatment, partly developed by CHOP, eliminates the need for regular infusions while preventing the ...

It’s now possible to treat inherited blood diseases, such as sickle cell disease, with gene editing. Blood stem cells are extracted from the patient, modified, and infused back into their bone ...

Gene therapy is linked to better nonmotor outcomes in spinal muscular atrophy type 1 compared to first-line nusinersen, new ...

University of Bristol researchers have discovered a VEGF-C gene therapy that protects the kidneys in people with type 1 ...

Gene therapy delivering protective vascular endothelial growth factor C into kidney filters reduced early diabetic kidney ...

Liver cells are indispensable for research—for drug testing, to better understand diseases such as hepatitis, fatty liver, cirrhosis, or liver cancer and for development of future cell therapies.

Long-term follow-up shows 95% success rate, no serious complications in largest ADA-SCID gene therapy study to date ...

A single one-time gene therapy could free patients with α-thalassemia, a rare and debilitating blood disorder, from the burden of lifelong transfusions. A single one-time gene therapy could free ...

A new gene therapy approach aimed at protecting people with type 1 diabetes from developing diabetic kidney disease—a serious and common complication of the condition, has shown promising results in a ...

An increasing demand for gene therapies is focusing the industry on enhancing production capabilities, transitioning from ...

New research found that gene therapy for children with "bubble boy disease," or SCID, was successful in 95% of trial ...