TSAI LAB
St. Jude Children's Research Hospital
Developing genome editing technologies as safe and effective therapies for human genetic conditions such as sickle cell disease.
Our Research
In the Tsai lab, our long-term goal is to advance safe and effective genome editing technologies to treat hematological conditions from bench to the bedside. Our current projects include:
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Protein engineering of CRISPR-Cas genome editors with improved activity, specificity, and precise control of DNA repair outcomes.
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Understanding safety of genome editors, including defining their genome-wide activities and interpreting the biological consequences.
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Advancing autologous genome edited hematopoietic stem cells for treatment of patients with sickle cell disease and bone marrow failure disorders to clinical trials at St. Jude.
We are grateful for support from the NIH Common Fund Somatic Cell Genome Editing Consortium, the Doris Duke Charitable Foundation, and St. Jude/ALSAC.
Select Publications
News
July 2026
CHANCE-seq paper accepted to publish in Nature
July 2026
DRIVE team, led by Dr. Tsai, awarded ARPA-H THRIVE $28.5M federal grant to advance in vivo base editing medicines for patients with bone marrow failure disorders.
January 2026
CHANGE-seq-BE paper published in Nature Biotechnology.
November 2025
Shengdar recognized as one of Highly Cited Researchers in 2025
November 2024
Shengdar recognized as one of Highly Cited Researchers in 2024
November 2023
Shengdar recognized as one of Highly Cited Researchers in 2023
May 2023
Tsai lab and co-investigators awarded NIH Somatic Cell Genome Editing Consortium grants to optimize CHANGE-seq, GUIDE-seq, and gRNA sequencing for use in development of genome editing medicines.
December 2022
Shengdar promoted to Associate Member.
November 2022
Shengdar recognized as one of highly cited researchers in 2022.
December 2021
GUIDE-seq protocol paper published in Nature Protocols.
November 2021
Shengdar recognized on list of Highly Cited Researchers in 2021.
September 2021
Rachael starts in the lab as a lead researcher.
August 2021
Elizabeth starts in the lab as a researcher.
June 2021
Andrea awarded NIH F31 pre-doctoral fellowship.
November 2020
CHANGE-seq paper published in Nature Biotechnology.
September 2020
Tsai and Weiss labs awarded renewal of DDCF funded grant to advance SCD genome editing therapy.
July 2020
Abby joins the lab as a research technologist.
May 2020
Shengdar honored by 2020 American Society of Gene and Cell Therapy Outstanding New Investigator Award.
March 2020
Congratulations to Andrea for passing her qualifying exams and becoming a PhD candidate.
October 2019
Kasey awarded Diggs postdoctoral fellowship