Author

Bazilco M.J. Simon

Gladstone Institutes

Listed by name: these papers carry no ORCID for this author, so different people with the same name may be mixed together.

Biology Disciplines

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Genetics· 1

Signature Assays, Organisms & Methods

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Assay:CRISPR screen· 1 Research Focus:drug response / drug discovery· 1 Approach:experimental assay· 1 Organism:human· 1

Frequent Co-Authors

Abin AbrahamiD· 1 shared Bruce R. ConkliniD· 1 shared Carissa M. Feliciano· 1 shared Chiara B.E. Marley· 1 shared Dylan Steury· 1 shared Erin N. GilbertsoniD· 1 shared Grace D. RameyiD· 1 shared Hannah L. Watry· 1 shared

Activity by Year

2025-2025 · peak 1/yr

Co-Author Collaboration Network

10 top collaborators · 45 mutual co-author links

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1 shared paperGenetics

Abin AbrahamORCID

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1 paper

Recent·Most cited

Haplotype editing with CRISPR/Cas9 as a therapeutic approach for dominant-negative missense mutations in NEFL

Poorvi H. Dua, Bazilco M.J. Simon, Chiara B.E. Marley, Carissa M. Feliciano, Hannah L. Watry, Quinn T. Cowan, Dylan Steury, Abin Abraham, Erin N. Gilbertson, Grace D. Ramey, John A. Capra, Bruce R. Conklin, Luke M. Judge

This paper demonstrates haplotype editing with CRISPR/Cas9 to target NEFL missense mutations in iPSC-derived motor neurons, showing therapeutic potential and challenges in disrupting mutant allele expression.

Molecular Therapy · Nov 20, 2025Peer-reviewedGenetics