Advancing human hematopoietic stem cell therapies: from sickle cell disease to personalized treatments for bone marrow failure disorders

Monday, September 22, 2025 at 12:30 pm US EDT

Dr. Shengdar Tsai will describe progress in our efforts to advanced personalized genome editing therapies for pediatric patients with bone marrow failure at St. Jude Children's Research Hospital, as part of a blue sky project called PARADIGM (Partnership to Advance Development of Individualized Genomic Medicines). 

Dr. Tsai is an Associate Member in the Department of Hematology at St. Jude Children’s Research Hospital. His lab’s research focuses on developing genome editing technologies for therapeutics, with a special interest in editing human HSCs for treatment of hemoglobinopathies such as sickle cell disease and T-cells for cancer immunotherapy. In 2020, he was chosen as one of the American Society for Gene and Cell Therapy Outstanding New Investigators.

His group has recently developed CHANGE-seq, a state-of-the-art, sensitive, unbiased, high-throughput method for defining the genome-wide activity of genome editors (Lazzarotto et al. Nature Biotechnology 2020). Previously, he has led the development of methods for high-throughput genome editing with TALENs (Reyon and Tsai et al. Nature Biotechnology 2012), CRISPR-Cas genome editors with improved specificity by dimerization (Tsai et al. Nature Biotechnology 2014), widely adopted methods to define the genome-wide specificity of CRISPR-Cas nucleases such as GUIDE-seq (Tsai and Zheng et al. Nature Biotechnology 2015) and CIRCLE-seq (Tsai et al. Nature Methods 2017 and Lazzarotto et al. Nature Protocols 2018).

Dr. Tsai completed a postdoctoral fellowship at Massachusetts General Hospital & Harvard Medical School, Ph.D. in Functional Genomics and M.S. in Bioinformatics from North Carolina State University, and B.S. from the University of Michigan.

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