Gene Therapy | CHOP Research Institute

Gene Therapy

Published on
Feb 6, 2023
Two scientists with unique skill sets are developing an alternative method for gene therapy delivery with support from a CGTC Seed Grant.

Dr. Kadauke is the associate director of the Cell and Gene Therapy Laboratory at CHOP and the medical director of the Cell and Gene Therapy Informatics team. His research focuses on developing novel ways to manufacture cell therapies with the goal of accelerating and broadening patient access to these life-saving therapies.

kadaukes [at]
Published on
Oct 14, 2022
Read the latest findings and new grants from Children’s Hospital of Philadelphia researchers.

Our team at the Congenital Hyperinsulinism Center at CHOP is working on a research study to determine whether an experimental gene therapy product, DTX401, is safe and effective in people with glycogen storage disease type Ia (GSDIa)

Published on
Sep 9, 2022
In this Q&A, hematologist Dr. Kwiatkowski discusses the Food and Drug Administration approval of beti-cel (Zynteglo®), a gene therapy for beta thalassemia.
Published on
Jun 24, 2022
This week’s In the News features endocrinology research, news on gene therapy in blood disorders, CAR T-cell therapy, and more.
Published on
May 19, 2022
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.
Published on
May 12, 2022
CHOP recognized gene therapy pioneer, Katherine High, MD, with Children’s Hospital of Philadelphia’s Gold Medal Award.
Published on
Mar 4, 2022
Learn about novel findings and new funding from our Children’s Hospital of Philadelphia researchers.
Published on
Nov 19, 2021
Dr. Lindsey George and colleagues report safe and sustained expression following hemophilia A gene therapy.