Viral Vectors | CHOP Research Institute
 

Viral Vectors

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

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)

Building on Nature's Delivery System to Advance Gene Therapy

Published on
Nov 5, 2021
in Cornerstone Blog
Raymond G. Perelman Center for Cellular and Molecular Therapeutics
The Clinical Vector Core, an award-winning, state-of-the-art, multi-product GMP manufacturing facility, supports the growing field of gene therapy.

Tunable Switch Via Oral Small Molecule Controls Gene Therapy Expression Levels

Published on
Jul 26, 2021
in Cornerstone Blog
Tunable Switch Via Oral Small Molecule Controls Gene Therapy Expression Levels Oral Small Molecule Helps to Fine-Tune Gene Therapy Expression
Children’s Hospital of Philadelphia scientists developed a tunable switch system to control levels of gene therapy expression.

Raymond G. Perelman Center for Cellular and Molecular Therapeutics

Raymond G. Perelman Center for Cellular and Molecular Therapeutics

Understanding, developing, and applying gene and related cell and nucleic acid therapies to find cures for debilitating disorders.

Protecting the Brain Against Alzheimer’s: Study to Advance Novel Gene Therapy

Published on
Nov 13, 2019
in Cornerstone Blog
Beverly Davidson, PhD
Beverly Davidson, PhD, director of the Center for Cellular and Molecular Therapeutics, is advancing work on a gene therapy for Alzheimer’s.

Realizing the Promise of Gene Therapy: Q&A With Clinical Vector Core Directors

Published on
Nov 6, 2018
in Cornerstone Blog
Johannes (Han) van der Loo, PhD, and Olga Zelenaia, PhD
Meet two of the scientists behind the game-changing gene therapies created by the Research Institute's Clinical Vector Core.

The Science Behind Gene Therapy: Creating Breakthroughs One Gene at a Time

Published on
Oct 8, 2018
in Cornerstone Blog
Gene therapy
An infographic and article look at the science behind gene therapy, challenges faced by the field, and the road ahead.