Gene Therapy | CHOP Research Institute
 

Gene Therapy

ASCGT 26th Annual Meeting Well-Attended by CHOP Scientists

Published on
May 18, 2023
in Cornerstone Blog
ASCGT 26th Annual Meeting Well-Attended by CHOP Scientists
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.

Optimizing Vector Delivery to Reduce Risk: Q&A With Beverly Davidson, PhD, and Misun Hwang, MD

Published on
Feb 6, 2023
in Cornerstone Blog
Gene Therapy and Radiology Team Up to Optimize Vector Delivery
Two scientists with unique skill sets are developing an alternative method for gene therapy delivery with support from a CGTC Seed Grant.
Stephan Kadauke, MD, PhD
Stephan Kadauke

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.

E-mail:
kadaukes [at] chop.edu

UCART22

UCART22

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

In the News: CureARS, Pigment Production, TBCK

Published on
Oct 14, 2022
in Cornerstone Blog
In the News
Read the latest findings and new grants from Children’s Hospital of Philadelphia researchers.

DTX401 Gene Therapy in Glycogen Storage Disease (GSD) Type Ia

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)

First Potentially Curative Gene Therapy for Beta Thalassemia: Q&A with Janet Kwiatkowski, MD, MSCE

Published on
Sep 9, 2022
in Cornerstone Blog
Q&A With Janet Kwiatkowski, MD, MSCE
In this Q&A, hematologist Dr. Kwiatkowski discusses the Food and Drug Administration approval of beti-cel (Zynteglo®), a gene therapy for beta thalassemia.

In the News: Featured Research, Tonsillectomy, Gene Therapy, CAR T-Cell Therapy

Published on
Jun 24, 2022
in Cornerstone Blog
In the News
This week’s In the News features endocrinology research, news on gene therapy in blood disorders, CAR T-cell therapy, and more.

Celebrating the Unsung Heroes of Research: The Ninth Annual CRC RE@CH Awards

Published on
May 19, 2022
in Cornerstone Blog
The Ninth Annual CRC RE@CH Award Winners Named
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.

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.