Clinical In Vivo Gene Therapy News & Announcements

We tallied up the most popular Cornerstone blog stories of 2024 based on data across our CHOP Research social media platforms.

Symposium highlights AAV gene therapy best practices for children, CHOP researchers develop novel nanopore platform, and more In the News

CHOP researchers developed a factor VIII variant that led to safe and long-lasting therapeutic results in preclinical models.

The CGTC helps to accelerate clinical trials, training, and research, enabling CHOP to remain a global leader in the space.

Using CRISPR/Cas9 gene editing in an early-stage clinical trial, researchers showed improved vision for a severe inherited form of blindness.

Decades ago, Katherine A. High, MD, and colleagues began an unprecedented journey to develop a new kind of treatment for hemophilia B.

As the administrative manager of the Clinical In Vivo Gene Therapy (CIGT) group, Sarah McCague manages a team of CRCs who are critical liaisons between families and the hospital.

This milestone represents an important step in treatment options for patients with hereditary hearing loss caused by genetic mutations.

CHOP's NoT Bleeding Program will conduct clinical trials to develop the next generation of therapeutics for hemophilia A, B, and other bleeding disorders.

Children’s Hospital of Philadelphia is the lead study site for the development of a gene therapy for multiple sulfatase deficiency.

The Clinical In Vivo Gene Therapy group supports CHOP’s continued leadership in the in vivo gene therapy space.

The FDA has granted accelerated approval of the first gene therapy for Duchenne muscular dystrophy — a major win for those affected by the debilitating disorder, CHOP experts say.

The Ninth Annual CRC RE@CH Awards celebrated all clinical research staff at CHOP for their contributions to the research community with a spotlight on two honorees.

Dr. Lindsey George and colleagues report safe and sustained expression following hemophilia A gene therapy.

We’re celebrating our research hero, Jaxson, and his super mom, Megan, this Mother’s Day. Thank you for making time for science.

An infographic and article look at the science behind gene therapy, challenges faced by the field, and the road ahead.