Author

Varun Katta

St. Jude Children's Research HospitalORCID 0000-0002-4565-7335Unclaimed ORCID profile · follow to get notified of new preprints

Biology Disciplines

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Genomics· 3 Genetics· 2

Signature Assays, Organisms & Methods

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Organism:human· 4 Assay:CRISPR screen· 3 Approach:experimental assay· 3 Research Focus:differential expression· 2 Research Focus:gene regulatory network inference· 2 Approach:statistical model· 2 Research Focus:variant calling· 2 Assay:whole-genome sequencing· 2

Frequent Co-Authors

Shengdar Q. TsaiiD· 5 shared Yichao LiiD· 4 shared Cícera Regina LazzarottoiD· 3 shared GaHyun LeeiD· 3 shared Elizabeth Urbina· 2 shared Garret ManqueniD· 2 shared Rachael K. WoodiD· 2 shared Yong ChengiD· 2 shared

Activity by Year

2022-2025 · peak 2/yr

Co-Author Collaboration Network

10 top collaborators · 42 mutual co-author links

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5 shared papersGenomics

Shengdar Q. TsaiORCID

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Author profile →

5 papers

Recent·Most cited

Brain-wide Genome Editing via STEP-RNPs for Treatment of Angelman Syndrome

Xiaona Lu, Ying Xie, Youmei Bao, Jiang Yu, Zefeng Wang, Jiali Fan, Wendy C. Sheu, Gretchen Long, Zewei Tu, Jie Tong, Garret Manquen, Yichao Li, Varun Katta, Ryan Dz-Wei Chow, Binfan Chen, , Jiangbing Zhou

This paper develops a non-viral genome-editing method called STEP-RNPs that effectively treats Angelman syndrome in mice and human neurons, showing functional genetic rescue without off-target effects.

bioRxiv · Nov 13, 2025 · PreprintGenetics

Frequent, context-dependent effects of human genetic variation on Cas9 activity revealed by population-scale GUIDE-seq-2 and deep combinatorial CHANCE-seq profiling

Ashley R. Flory, Cícera Regina Lazzarotto, Yichao Li, Jacqueline Chyr, Muyu Yang, Varun Katta, Elizabeth Urbina, GaHyun Lee, Rachael K. Wood, Azusa Matsubara, Claire Greene Whitfield, Sara R. Rashkin, Jian Ma, Yong Cheng, Shengdar Q. Tsai

The paper uses population-scale GUIDE-seq-2 and CHANCE-seq to analyze how genetic variants affect Cas9 activity, finding frequent context-dependent effects on off-target sites.

bioRxiv · Feb 10, 2025 · PreprintGenomics

Development and IND-enabling studies of a novel Cas9 genome-edited autologous CD34+ cell therapy to induce fetal hemoglobin for sickle cell disease

Varun Katta, Kiera O’Keefe, Yichao Li, Thiyagaraj Mayuranathan, Cícera Regina Lazzarotto, Rachael K. Wood, Rachel M. Levine, Alicia Powers, Kalin D. Mayberry, Garret Manquen, Yu Yao, Jingjing Zhang, Yoonjeong Jang, Nikitha Nimmagadda, Erin Dempsey, , Shengdar Q. Tsai

This study develops a genome-edited autologous CD34+ cell therapy to induce fetal hemoglobin for sickle cell disease, showing efficient editing, therapeutic HbF levels, and no off-target effects.

Molecular Therapy · Jul 30, 2024Peer-reviewedGenomics

CHANGE-seq-BE enables simultaneously sensitive and unbiased in vitro profiling of base editor genome-wide activity

Cícera Regina Lazzarotto, Varun Katta, Yichao Li, Elizabeth Urbina, GaHyun Lee, Shengdar Q. Tsai

The paper introduces CHANGE-seq-BE, a method for sensitive and unbiased profiling of base editor off-target effects in vitro.

bioRxiv · Mar 28, 2024 · PreprintGenomics

Human genetic diversity modifies therapeutic gene editing off-target potential

Samuele Cancellieri, Jing Zeng, Linda Yingqi Lin, Manuel Tognon, My Anh Nguyen, Jiecong Lin, Nicola Bombieri, Stacy A. Maitland, Marioara-Felicia Ciuculescu, Varun Katta, Shengdar Q. Tsai, Myriam A. Armant, Scot A. Wolfe, Rosalba Giugno, Daniel E. Bauer, Luca Pinello

The paper develops CRISPRme, a tool that integrates genetic diversity data to assess off-target risks of gene editing, finding allele-specific off-target effects in clinical gRNAs.

Nature Genetics · Dec 15, 2022Peer-reviewedGenetics