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

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Monogenic Diseases

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

Safety of Gene Therapy

Overview of Viral Vector Shedding

Vector shedding is the release of virus-based gene therapy products from the patient through one or all of the following routes: excreta (faeces), secreta (urine, saliva, nasopharyngeal fluids, etc.), and skin (pustule, sores, wounds)1

Practical Considerations

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

Gene Inhibition Therapy

Endogenous RNAi Pathway

The video below will provide an overview of the endogenous or naturally occurring RNA interference (or RNAi) pathway.

STEERING COMMITTEE

Meet our multidisciplinary, internationally renowned steering committee of experts:

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Chris Jenkins, PhD, MPH
Chairperson
Principal Partner and Chief Gene Therapy Biosafety Officer at Clinical Biosafety Services

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Diana Bharucha-Goebel
Director of the Muscular Dystrophy Association Care Center and Assistant Professor in the Division of Neurology
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Arthur Burghes
Professor at Wexner Medical Center College of Medicine in the Department of Biological Chemistry and Pharmacology
James Dowling, MD, PhD, FANA
Professor of Pediatrics and Molecular Genetics at the University of Toronto

Uta Griesenbach, DIPL-BIOL, PhD
Professor in Molecular Medicine at the National Heart and Lung Institute at Imperial College London

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Kristi Jones, PhD
Clinical Associate Professor of Pediatrics, Child Health and Genetic Medicine at The University of Sydney, and Co-Head of the Department of Clinical Genetics at The Children’s Hospital at Westmead

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John Petrich
Director Investigational Drug Services at the Cleveland Clinic