Results for Adeno-associated Virus Gene Transfer

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Where Discovery Leads: Building on Nature's Delivery System to Advance Gene Therapy

Published on
Nov 5, 2021
in Cornerstone Blog
Raymond G. Perelman Center for Cellular and Molecular Therapeutics
The Clinical Vector Core, an award-winning, state-of-the-art, multi-product GMP manufacturing facility, supports the growing field of gene therapy.

Grant Awarded to Study Gene Therapy for Disorders With Severe Brain Disease

Published on
Dec 4, 2019
in Cornerstone Blog
John Wolfe, VMD, PhD, is studying gene therapy methods to correct the brain disease of lysosomal storage disorders.
John Wolfe, VMD, PhD, receives grant to study gene therapy treatment for lysosomal storage disorders with severe disease of the brain.

In the News: Advancing Gene Therapy, CSF Tests, Managing Vascular Anomalies, New Clinical Futures Director

Published on
Aug 9, 2019
in Cornerstone Blog
CHOP Research In the News
Heat waves, drenching rainstorms, and vacation escapes can’t stop our investigators from their continued advancement of scientific discovery.

The Science Behind Gene Therapy: Creating Breakthroughs One Gene at a Time

Published on
Oct 8, 2018
in Cornerstone Blog
Gene therapy
An infographic and article look at the science behind gene therapy, challenges faced by the field, and the road ahead.

Pioneering Novel Therapies for Hemophilia

Published on
Sep 20, 2018
in Cornerstone Blog
hemophilia
Two exciting lines of research build on what we know about hemophilia to improve existing treatments.

Vaccinating Against Diseases, Without a Vaccine

Published on
Mar 18, 2015
in Cornerstone Blog
Last month, Dr. Farzan published a study in Nature that describes his team’s creation of a new molecule that prevents monkeys from being infected with simian/human immunodeficiency virus.

Clinical In Vivo Gene Therapy

Clinical In Vivo Gene Therapy

The Clinical In Vivo Gene Therapy group supports the development and conduct of in vivo gene and molecular therapy trials at CHOP, as well as the implementation of these novel molecular therapies into the clinic post licensure.

Gene Therapy Trial for Hemophilia A

Gene Therapy Trial for Hemophilia A

This clinical trial study uses a modified adeno-associated virus (AAV) to carry the human factor VIII gene to the liver.

Clinical Vector Core

Clinical Vector Core

The Raymond G. Perelman Center for Cellular and Molecular Therapeutics has established state-of-the-art Current Good Manufacturing Practices (cGMP) clinical vector manufacturing suites for adeno-associated virus (AAV) and lentiviral (LV) vectors.

Novel Therapeutics for Bleeding Disorders

Novel Therapeutics for Bleeding Disorders

Combining our proven clinical expertise in hemophilia gene therapy and care of bleeding disorders with our research expertise to solidify Children’s Hospital of Philadelphia as the premier center for the therapeutic development and advanced clinical care of inherited bleeding disorders.

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