Adeno-associated Viral Vectors | CHOP Research Institute

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)

Cornerstone Blog

Research Shows Multiyear Factor VIII Expression After Hemophilia A Gene Therapy

Published on

Nov 19, 2021

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.

Sabatino Laboratory

The Sabatino Lab is focused on the inherited bleeding disorder, hemophilia. Our research interests include the study of variants of coagulation factor VIII to understand the biochemical properties of these proteins and to identify novel variants with enhanced function, and the development of gene-based therapeutic treatment approaches.

Cornerstone Blog

Advancing Gene Therapy, CSF Tests, Managing Vascular Anomalies, New Clinical Futures Director

Published on

Aug 9, 2019

Heat waves, drenching rainstorms, and vacation escapes can’t stop our investigators from their continued advancement of scientific discovery.

Denise E. Sabatino, PhD

The research in the Sabatino Laboratory is focused on hemophilia, an inherited bleeding disorder. The interests of the laboratory include the study of variants of coagulation factor VIII to understand the biochemical properties of these proteins and to identify novel variants with enhanced function, and the development of gene-based therapeutic approaches for treating hemophilia.

E-mail:

sabatinod [at] chop.edu