Tag: Gene Therapy Drugs

Wexner Medical Center among first in nation to administer new gene therapy for ALS

sarah JonasFeature

The Ohio State University Wexner Medical Center is one of the first nationwide to administer a targeted gene therapy for patients with a specific form of amyotrophic lateral sclerosis (ALS), a neurodegenerative disease that affects the cells in the brain and spine.

Children’s Hospital of Philadelphia Awards Gold Medal to Dr. Katherine High, Gene Therapy Pioneer

sarah JonasAnnouncement

In an honor reserved for only the most significant achievements in advancing children’s health, Children’s Hospital of Philadelphia (CHOP) has awarded its Gold Medal to Katherine High, MD, a gene therapy pioneer and one of the lead developers of the first in vivo gene therapy approved by the Food and Drug Administration (FDA). Bestowed only 12 times in CHOP’s 167-year history, the Gold Medal highlights Dr. High’s groundbreaking discoveries at CHOP, which led to a gene therapy treatment for a rare form of inherited blindness and advanced gene therapy for hemophilia to late-stage testing.

Children’s Hospital of Philadelphia Researchers Develop “Dimmer Switch” to Help Control Gene Therapy

sarah JonasResearch Results

In a major advancement in the field of gene therapy for rare and devastating diseases, researchers at Children’s Hospital of Philadelphia (CHOP) have developed a “dimmer switch” system that can control levels of proteins expressed from gene therapy vectors. The system is based on alternative RNA splicing using an orally available small molecule and works effectively in tissues throughout the body, including the brain. The first research regarding this innovation was published today in the journal Nature.

Scientists Aim Gene-Targeting Breakthrough Against COVID-19

sarah JonasFeature

Scientists at Berkeley Lab and Stanford have joined forces to aim a gene-targeting, antiviral agent called PAC-MAN against COVID-19.