CT researchers explore gene therapy that shows promise to slow aging, fight cancer

With promise to slow aging and cure some types of cancer, a new gene therapy presents cost and access hurdles for CT patients ...
Hosted on MSN

Scientists discover new approach to gene therapy

Researchers have found a promising new method for gene therapy. They successfully restarted inactive genes by bringing them closer to genetic switches on the DNA called enhancers. The intermediate ...
The American Journal of Managed Care

Switching to Gene Therapy After Nusinersen or Risdiplam May Benefit Patients With SMA

Onasemnogene abeparvovec gene therapy post-nusinersen or risdiplam showed meaningful motor improvements in children with SMA, with a manageable safety profile. The study cohort included older, heavier ...
Forbes

The Triumphs And Trials Of Duchenne Gene Therapy

Forbes contributors publish independent expert analyses and insights. William A. Haseltine, Ph.D., covers genomics and regenerative medicine Today, the world stands at a crossroads in genetic medicine ...
Ophthalmology Times

First patient dosed in phase I/II trial of complement-targeting gene therapy for geographic atrophy

A first-in-human clinical trial evaluating an investigational gene therapy targeting complement-mediated pathways in ...

FDA approves gene therapy for severe leukocyte adhesion deficiency-I, a rare immune disorder

Dr. Donald Kohn has been developing gene therapies for rare pediatric immune disorders for over 30 years. This week, his role in a clinical trial has culminated in the first-ever U.S. Food and Drug ...
NPR

Gene therapy is transforming lives, but for many Americans it's hard to reach

Dustin Vidrine's sight is disappearing — shrinking, really. He has retinitis pigmentosa, a rare, inherited eye disease that runs in his family. By the time he was in his 20s, he noticed that his ...
The American Journal of Managed Care

Gene Therapy

Progress and Challenges With Gene Therapy in Hereditary Hearing Loss Vectors, both viral and non-viral, continue to further progress gene therapy for a variety of disease states. With further ...
Forbes

Gene Editing Or Gene Silencing: The Therapies Poised To Transform Medicine

Revolutions in gene therapy are rapidly changing the landscape of modern medicine. Revolutions in gene therapy are rapidly changing the landscape of modern medicine, forcing society and science alike ...
Hosted on MSN

Gene therapy for hereditary spastic paraplegia hits proof-of-principle milestone

There is no cure for the rare disease Hereditary Spastic Paraplegia (HSP), but researchers from Drexel University's College of Medicine and the UMass Chan Medical School have achieved ...