Half of all children with rare diseases will never see an available treatment reach them not because the science isn’t ...

Researchers at The Ohio State University Wexner Medical Center are using novel nonviral gene therapy technology to gain ...

Researchers from Drexel University’s College of Arts and Sciences and College of Medicine have found a potential new ...

Heart diseases are still the number one cause of death worldwide. But scientists are trying to change that... | Genetics And ...

The post Gene-Therapy Treatment Of Infant With Rare Genetic Condition Successful In World First first on TwistedSifter.

How Does Gene Therapy for Hemophilia Work? Gene therapy, which is now FDA-approved for hemophilia A and hemophilia B, deliver genes for factor VIII or IX via adeno-associated viruses.

Children with spinal muscular atrophy (SMA) showed motor function improvements after switching to onasemnogene abeparvovec ...

Gene therapy appears to be a promising approach for a subset of genetic deafness, although challenges remain with development and manufacturing.

The now multibillion dollar industry just in the Columbus area has had a presence in the region for the last decade.

Gene therapy can be less effective in women—and my research in mice brings us one step closer to understanding why by Alison Clare, The Conversation edited by Gaby Clark, reviewed by Andrew Zinin ...