Gene Therapy | CHOP Research Institute

Gene Therapy

Published on
May 18, 2023
Scientists from Children’s Hospital of Philadelphia attend the American Society of Cell & Gene Therapy Annual Meeting to share their expertise through posters, panel discussions, and workshops.
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]

The purpose of this study is to determine the safety and tolerability of an experimental form of therapy, known as UCART22 ("study drug").

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.