Adeno-associated Viral Vectors | CHOP Research Institute
 

Adeno-associated Viral Vectors

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)

Research Shows Multiyear Factor VIII Expression After Hemophilia A Gene Therapy

Published on
Nov 19, 2021
in Cornerstone Blog
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

Sabatino Lab

The research in the Sabatino Laboratory is focused on the inherited bleeding disorder, hemophilia.

Arruda Laboratory

Arruda Lab

The development of gene-based strategies for the treatment of bleeding and thrombotic diseases is at the heart of research in the Arruda Lab. Working collaboratively, the lab team and their colleagues have carried out early-phase clinical studies on adeno-associated viral vectors for the treatment of severe hemophilia B.

In the News: Advancing Gene Therapy, CSF Tests, Managing Vascular Anomalies, New Clinical Futures Director

Published on
Aug 9, 2019
in Cornerstone Blog
CHOP Research In the News
Heat waves, drenching rainstorms, and vacation escapes can’t stop our investigators from their continued advancement of scientific discovery.
Denise E. Sabatino, PhD
Denise E. Sabatino

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