Rachael K. Wood

In vivo Treatment of a Severe Vascular Disease via a Bespoke CRISPR-Cas9 Base Editor

This paper develops a customized CRISPR-Cas9 base editor to correct the ACTA2 R179H mutation in a mouse model of MSMDS, improving survival and rescuing vascular and neurological phenotypes.

Frequent, context-dependent effects of human genetic variation on Cas9 activity revealed by population-scale GUIDE-seq-2 and deep combinatorial CHANCE-seq profiling

The paper uses population-scale GUIDE-seq-2 and CHANCE-seq to analyze how genetic variants affect Cas9 activity, finding frequent context-dependent effects on off-target sites.

Development and IND-enabling studies of a novel Cas9 genome-edited autologous CD34+ cell therapy to induce fetal hemoglobin for sickle cell disease

This study develops a genome-edited autologous CD34+ cell therapy to induce fetal hemoglobin for sickle cell disease, showing efficient editing, therapeutic HbF levels, and no off-target effects.