Author
University of California, San FranciscoORCID 0000-0001-5321-131XUnclaimed ORCID profile · follow to get notified of new preprints
3 papers
Recent·Most cited
Akshita Saxena, Grace D. Ramey, John A. Capra…·Bria L Macklin
EXCAVATE-HT identifies targetable genomic variants for allele-specific CRISPR editing by analyzing population data and generating gRNA libraries.
Grace D. Ramey, Quinn T. Cowan, Akshita Saxena…·John A. Capra
This paper identifies therapeutic opportunities for over 500 genes with dominant and dispensable disease alleles by enabling mutation-agnostic gene therapy through allele-specific targeting.
Poorvi H. Dua, Bazilco M.J. Simon, Chiara B.E. Marley…·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.