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ISCT Webinar 2020: Gene Therapy and Gene Editing for Hemoglobinopathies

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Gene Therapy and Gene Editing for Hemoglobinopathies: The Basics, The Present, and The Future

An ISCT Immuno & Gene Therapy Committee Webinar

Sponsored by ISCT

Event Date
Thursday, February 6, 2020
09:00 PDT, 12:00 EDT, 18:00 CET


About the Webinar
The ongoing progression of gene therapies towards clinical use has led to an increased number of clinical trials in recent years and global discourse around the therapeutic potential, ethical considerations and future application of gene therapy and gene editing. As a result, there is an increased need for patient resources that provide information and insight into these novel therapies.

Developed by the ISCT Immuno-Gene Therapy Committee as a resource for patient advocacy groups, patients and their families, this webinar will define gene therapy and outline the mechanism of action and current status of gene therapies for hemoglobinopathies, such as sickle cell disease and thalassemia. To close the session the future of gene therapy and gene editing will be discussed, and key considerations identified that can help ensure the safe and effective utilization and overall advancement of such therapeutic products.

Andrew Fesnak, MD
University of Pennsylvania
United States

Sung-Yun Pai, MD
Boston Children’s Hospital
United States


Allistair Abraham, MD
Children’s National Hospital
United States

Erica Esrick, MD
Boston Children’s Hospital;
Dana-Farber Cancer Institute; Harvard Medical School
United States

Sandeep Soni, MD
Stanford University
United States


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