Ross C. Wilson

Chemically modified CRISPR enzymes for multi-organ genome editing in vivo

Delivery remains the main obstacle to the development of in vivo genome editing therapies. CRISPR ribonucleoproteins confer high editing activity with transient exposure but lack intrinsic cell entry and targeting. Here we introduce PERCEPT, a delivery platform featuring reversible, covalent modification of CRISPR enzy...

Peptide-mediated CRISPR delivery: From dish to bloodstream.

CRISPR-based therapeutics have advanced rapidly, but clinical translation remains bottlenecked by delivery. Standard carriers like viral vectors and lipid nanoparticles (LNPs) face challenges regarding cargo capacity, immunogenicity, and restricted tissue tropism. Peptide-mediated delivery platforms offer a highly tuna...

LNP-mediated BCL11A Editing Corrects Sickling Phenotypes and Preserves HSPC Fitness Compared to Electroporation

Although RNP electroporation achieves higher CRISPR editing rates, it reduces HSPC recovery and RBC differentiation; conversely, mRNA-LNP delivery achieves modest rates while preserving HSPC fitness, progenitor expansion, and RBC enucleation efficiencies.

LNP-mediated BCL11A Editing Corrects Sickling Phenotypes and Preserves HSPC Fitness Compared to Electroporation