Author
Massachusetts Eye and Ear InfirmaryORCID 0009-0002-0903-6475Unclaimed ORCID profile · follow to get notified of new preprints
3 papers
Recent·Most cited
Bryan L. Peacker, Kuan‐Hung Lin, Amy Lam…·Amy J. Wagers
This study demonstrates CRISPR-HDR efficiency in postnatal cardiac, skeletal muscle, and muscle stem cells, showing developmental stage-dependent HDR rates and successful correction of a Dmd mutation in mdx mice.
Samantha DeRosa, Monica Salani, Sierra Smith…·Yulia Grishchuk
This study demonstrates that AAV-mediated MCOLN1 gene transfer improves motor function and reduces brain pathology in a mouse model of mucolipidosis IV.
Suhani Vora, Jenny Yeuk‐Ki Cheng, Ru Xiao…·George McDonald Church
The paper designs a compact CRISPR-Cas9 activator suitable for AAV-mediated delivery and validates its transcriptional activation efficacy in multiple cell lines.