Author

Shota Shibata

Broad InstituteORCID 0000-0002-9131-5408Unclaimed ORCID profile · follow to get notified of new preprints

Biology Disciplines

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Signature Assays, Organisms & Methods

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Approach:experimental assay· 2 Research Focus:benchmarking· 1 Research Focus:drug response / drug discovery· 1 Organism:human· 1 Organism:mouse· 1

Frequent Co-Authors

James F. GusellaiD· 2 shared Kevin CorreiaiD· 2 shared Marcy E. MacDonald· 2 shared Marina V. KovalenkoiD· 2 shared Ricardo Mouro PintoiD· 2 shared Tammy GillisiD· 2 shared Vanessa Chantal WheeleriD· 2 shared Antonia G. Vitalo· 1 shared

Activity by Year

2024-2025 · peak 1/yr

Co-Author Collaboration Network

10 top collaborators · 43 mutual co-author links

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2 shared papersGenetics

James F. GusellaORCID

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2 papers

Recent·Most cited

Identification of genetic modifiers of Huntington's disease somatic CAG repeat instability by in vivo CRISPR-Cas9 genome editing

Ricardo Mouro Pinto, Ryan Murtha, António Azevedo, Cameron J. Douglas, Marina V. Kovalenko, Jessica Ulloa, Steven Crescenti, Zoe N. Burch, Esaria Oliver, Maheswaran Kesavan, Shota Shibata, Antonia G. Vitalo, Eduarda Mota‐Silva, Marion J. Riggs, Kevin Correia, , Vanessa Chantal Wheeler

The paper identifies genetic modifiers of Huntington's disease somatic CAG repeat instability using CRISPR-Cas9 in HD knock-in mice, revealing new therapeutic targets.

Nature Genetics · Jan 22, 2025Peer-reviewedGenetics

PMS1 as a target for splice modulation to prevent somatic CAG repeat expansion in Huntington's disease

Zachariah L. McLean, Dadi Gao, Kevin Correia, Jennie C. L. Roy, Shota Shibata, Iris N. Farnum, Zoe Valdepenas-Mellor, Marina V. Kovalenko, Manasa Rapuru, Elisabetta Morini, Jayla Ruliera, Tammy Gillis, Diane E. Lucente, Benjamin P. Kleinstiver, Jong‐Min Lee, , James F. Gusella

This study shows splice modulators like branaplam reduce HTT CAG repeat expansion by affecting PMS1 expression, suggesting PMS1 as a new therapeutic target for Huntington's disease.

Nature Communications · Apr 12, 2024Peer-reviewedGenetics