Gene Therapy | CHOP Research Institute
 

Gene Therapy

CIGT Advances CHOP's In Vivo Gene Therapy Leadership

Published on
Aug 29, 2023
in Cornerstone Blog
CIGT Advances CHOP's In Vivo Gene Therapy Leadership
The Clinical In Vivo Gene Therapy group supports CHOP’s continued leadership in the in vivo gene therapy space.

‘Medicine of the Future’: Delivering mRNA Gene Editing Tools In Vivo

Published on
Jul 31, 2023
in Cornerstone Blog
‘Medicine of the Future’ in Development at CHOP to Treat Blood Disorders
Researchers developed a proof-of-concept model for delivering gene editing tools to treat blood disorders, allowing for the modification of diseased blood cells directly within the body.

FDA Approves First Gene Therapy to Treat Duchenne Muscular Dystrophy

Published on
Jun 26, 2023
in Cornerstone Blog
FDA Approves First Gene Therapy to Treat Duchenne Muscular Dystrophy
The FDA has granted accelerated approval of the first gene therapy for Duchenne muscular dystrophy — a major win for those affected by the debilitating disorder, CHOP experts say.

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.