Results for Adeno-associated Viral Vectors

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Katherine High, MD, Awarded Esteemed CHOP Gold Medal

Published on
May 12, 2022
in Cornerstone Blog
Katherine High, MD, Awarded Esteemed CHOP Gold Medal
CHOP recognized gene therapy pioneer, Katherine High, MD, with Children’s Hospital of Philadelphia’s Gold Medal Award.

Research Shows Multiyear Factor VIII Expression After Hemophilia A Gene Therapy

Published on
Nov 19, 2021
in Cornerstone Blog
A one-time treatment with an adeno-associated viral vector demonstrated safe, sustained factor VIII expression for patients with hemophilia A.
Dr. Lindsey George and colleagues report safe and sustained expression following hemophilia A gene therapy.

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.

CHOP Cell and Gene Therapy Experts Elected to National Academy of Medicine

Published on
Oct 21, 2019
in Cornerstone Blog
Stephen A. Grupp and Beverly Davidson
Join us in congratulating cell and gene therapy experts Beverly Davidson, PhD, and Stephan Grupp, MD, PhD, newly elected to the National Academy of Medicine.

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.

Repairing DNA, One Cell at a Time

Published on
Jan 16, 2014
in Cornerstone Blog
High is one of the world’s leading experts in gene therapy, which has long been a “next big thing” in medicine: Take a person with a devastating genetic disease and replace their nonfunctional gene with a normal one — a cure built right into your DNA.

Research Vector Core

Research Vector Core

The Research Vector Core provides state-of-the-art technology support for investigators requiring viral-based vectors for gene transfer in basic research and pre-clinical studies.

Sabatino Laboratory

Sabatino Lab

The research in the Sabatino Laboratory is focused on the inherited bleeding disorder, hemophilia.

Samelson-Jones Laboratory

Samelson-Jones Lab

Our lab is interested in the hemostasis system, especially coagulation factor VIII and IX, which are deficient in the bleeding disorders hemophilia A and B. By studying these blood proteins, the lab team aims to improve therapies for children with these diseases including gene therapy.

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