Kathy J. Snow

The paper develops new mouse reporter models to detect genome editing events in vivo, including gene repair and CRISPR indels, and A-base editor activity.

The paper validates two genes, Cnot1 and Cdc42bpb, as causes of congenital diaphragmatic hernia in mice using patient-specific variants and shows distinct hernia phenotypes.

This study develops Neuro-PERC, a peptide-mediated CRISPR delivery method, enabling efficient in vivo neuronal editing in mice and pigs, and extending survival in a Huntington's disease model.

This paper maps the tissue tropism of ten AAV serotypes in mice after systemic delivery, identifying liver preference and unexpected transduction in adrenals, testes, and ovaries.

This study develops non-viral nanocapsules for in vivo CRISPR-Cas9 delivery to edit neuronal DNA in mice, achieving efficient and safe genome editing in specific neuron types.