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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:​

  • Protein engineering of CRISPR-Cas genome editors with improved activity, specificity, and precise control of DNA repair outcomes.

  • Understanding safety of genome editors, including defining their genome-wide activities and interpreting the biological consequences.

  • Advancing autologous genome edited hematopoietic stem cells for treatment of patients with sickle cell disease to a clinical trial 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.



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

July 2019

Michelle, Kiera, and Tiffany join the lab as research technologists.

June 2019

Andrea joins the lab as a graduate student.

October 2018

Kasey joins the lab as a post-doctoral fellow.

April 2018

Varun joins the lab as a senior research technologist.

September 2018

Tsai lab and co-investigators awarded NIH Somatic Cell Genome Editing grant to develop a novel T-cell platform to understand biological adverse effects of genome editing.

June 2017

CIRCLE-seq paper published in Nature Methods.

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