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Rare Disease : Innovations in Diagnostics and Therapeutics (part 3) : Sickle Cell Disease

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For this last series of lectures dedicated on Innovation in Diganostics and Therapeutics in Rare Disease, we have selected 2 lectures on Sickle Cell Disease.

These conferences have been given during a symposium organized by UCSF Innovation Ventures.





Curative Therapies for SCD

By Mark C. Walters, MD (Director of the Blood and Marrow Transplantation Program)

Mark C. Walters, MD is the Jordan Family Director of the Blood and Marrow Transplantation Program at UCSF Benioff Children’s Hospital, Oakland, and Professor of Pediatrics at UCSF School of Medicine. He is Program Director of the California Institute of Regenerative Medicine alpha stem cell clinic at UCSF. Dr. Walters received his A.B. with honors in Genetics from the University of California, Berkeley and his MD from the University of California, San Diego. He completed pediatric residency training at the University of Washington and hematology/oncology fellowship training at the University of Washington and the Fred Hutchinson Cancer Research Center in Seattle. He has been active in cooperative clinical transplantation trials and has led several NIH-supported investigations of hematopoietic cell transplantation for sickle cell anemia and thalassemia. He has a research interest in extending transplantation to young adults with hemoglobin disorders and other novel cellular therapies for hemoglobin disorders. Currently, research interests are focused on genomic editing and gene addition therapies as a strate- gy to extend curative therapy in all patients who inherit a clinically significant hemoglobinopathy.



Understanding and Addressing Health Disparities in SCD

By Marsha J. Treadwell, PhD, UCSF (UCSF Professor of Pediatrics, Hematology)

Marsha J. Treadwell is a Professor of Pediatrics at UCSF in the Division of Hematology and is a Jordan Fund Endowed Chair at UCSF Benioff Children’s Hospital

Oakland. She is Co-Chair of the Diversity, Equity, Inclusion and Anti-Racism Council for the UCSF Benioff Children’s Hospitals. Dr. Treadwell’s research integrates physical, behavioral and psychological processes, allowing for the identification of risk and resiliency factors and the development of more effective interventions for populations made vulnerable by systems in both high and low resource settings. Dr. Treadwell has expertise with measurement development, community engagement, conducting health care quality improvement projects, evaluating programs and assessing health-related quality of life for individuals and communities. Dr. Treadwell is Co- PI and Regional Director for the Pacific Sickle Cell Regional Collaborative. She is co-PI and Director for a Sickle Cell Disease Implementation Consortium (SCDIC) site, funded by the National Heart, Lung and Blood Institute. The goal of the SCDIC is to use implementation science to improve outcomes for adolescents and adults with sickle cell disease by improving understanding of barriers to care and utilization of evidence-based guidelines. Dr. Treadwell received her doctorate in clinical child psychology from the University of Washington in Seattle, WA and advanced training in clinical research from UCSF.

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