Concurrent Session: Balancing risks and outcomes; Gene therapy vs Transplant
Friday, September 8, 2023 08:00 AM - 09:00 AM
335 AB
Chair
Jaap Jan Boelens, MD, PhD, Transplantation and Cellular Therapies, MSKCC, USA
Speakers
Michael Keller, MD, Children's National, USA – Gene therapy and Transplant for Inborn Errors of Immunity: Successes and Hurdles
Akshay Sharma, MBBS, St. Jude Children's Research Hospital, USA – Gene Therapy and Transplant for Sickle Cell Disease
Troy Lund, MD, PhD, University of Minnesota Medical School – Gene Therapy vs Transplant Cerebral ALD
Gene therapy is entering the market; expectation is that more and more products will get approved. How do these products compare with BMT outcomes? Outcomes (LT and short term) comparison for PIDs, metabolic diseases and Hemoglobinopathies will be discussed.
Session Objecctives
Jaap Jan Boelens, MD, PhD, Transplantation and Cellular Therapies, MSKCC, USA
Speakers
Michael Keller, MD, Children's National, USA – Gene therapy and Transplant for Inborn Errors of Immunity: Successes and Hurdles
Akshay Sharma, MBBS, St. Jude Children's Research Hospital, USA – Gene Therapy and Transplant for Sickle Cell Disease
Troy Lund, MD, PhD, University of Minnesota Medical School – Gene Therapy vs Transplant Cerebral ALD
Gene therapy is entering the market; expectation is that more and more products will get approved. How do these products compare with BMT outcomes? Outcomes (LT and short term) comparison for PIDs, metabolic diseases and Hemoglobinopathies will be discussed.
Session Objecctives
- What are advantages and limitations of stem cell gene therapy for the various genetic diseases groups
- What are advantages and limitations of bone marrow transplantation for the various genetic diseases groups
- Risk - Benefit balance is different for the different genetic disease groups
Jaap Jan Boelens MD, PhD
Pediatric-Oncologist, Chief
Transplantation and Cellular Therapies, MSKCC
Chair
Pediatric-Oncologist, Chief
Transplantation and Cellular Therapies, MSKCC
Chair
Michael Keller
Associate Professor of Pediatrics, Division of Allergy & Immunology, Center for Cancer and Immunology Research, Director, Jeffrey Modell Diagnostic and Research Center, For Primary Immunodeficiencies
Children's National Hospital
Speaker
Associate Professor of Pediatrics, Division of Allergy & Immunology, Center for Cancer and Immunology Research, Director, Jeffrey Modell Diagnostic and Research Center, For Primary Immunodeficiencies
Children's National Hospital
Speaker
Troy Lund
Associate Professor, Department of Pediatrics
University of Minnesota Medical School
Speaker
Associate Professor, Department of Pediatrics
University of Minnesota Medical School
Speaker
