Welcome to the leading edge of gene therapy education

The mission of the GT Network is to provide healthcare professionals with high quality, scientific information about gene therapy

homepagedna.svg

MODULE 1

Overview of Gene Therapy

An overview of gene therapy and some of the key concepts in this field

MODULE 2

Monogenic Diseases

An overview of genetic abnormalities and current treatment strategies for monogenic diseases

MODULE 3

Safety of Gene Therapy

A review of the two types of immune responses in the context of two of the most widely studied viral vectors

MODULE 4

Practical Considerations

An overview of the biological risks and containment practices for use of AAV-based gene therapies

MODULE 5

Gene Replacement

One important approach to gene therapy is gene replacement. This video further explores this important concept.

MODULE 6

Current Therapeutics & Research

A review of the gene therapy development landscape and exploration of marketed agents

NEWS

STEERING COMMITTEE

Meet our multidisciplinary, internationally renowned steering committee of experts:

GTNSteering-goebel
Diana Bharucha-Goebel
Director of the Muscular Dystrophy Association Care Center and Assistant Professor in the Division of Neurology
GTNSteering-burghes
Arthur Burghes
Professor at Wexner Medical Center College of Medicine in the Department of Biological Chemistry and Pharmacology
GTNSteering-dastgir
Jahannaz Dastgir
Medical Director of the Pediatric Neuromuscular Medicine Program at Goryeb Children’s Hospital
GTNSteering-jenkins
Chris Jenkins
Principal Partner and Chief Gene Therapy Biosafety Officer at Clinical Biosafety Services
GTNSteering-kaufmann
Petra Kaufmann
Vice President of R&D Translational Medicine at AveXis
GTNSteering-mackenzie
Alex MacKenzie
Principal Investigator at the Children’s Hospital of Eastern Ontario
GTNSteering-petrich
John Petrich
Director Investigational Drug Services at the Cleveland Clinic
GTNSteering-pena
Loren del Mar Peña
Associate Professor of Pediatrics at the University of Cincinnati, Ohio