Donald B. Kohn

This study identifies synthesis errors in ssODNs used for HDR-mediated gene editing and shows they propagate into the genome, potentially causing disease alleles.

This paper develops lipid nanoparticles to deliver CRISPR/Cas9 for site-specific CFTR gene integration in CF airway cells, achieving 3-3.5% gene editing and 80% Cl- current restoration.

This study demonstrates that low levels of CFTR integration can restore chloride ion currents in human airway cells using optimized nonviral CRISPR-Cas9 RNP and double-stranded DNA donors.

This study generates TCR-null CAR T cells from iPSCs by controlling CAR signaling stages, resulting in naive-like cells with enhanced cytotoxicity.

This paper demonstrates successful in vivo editing of ApoE4 to E3 in Alzheimer's model mice using synthetic exosome-delivered CRISPR, achieving low but detectable editing efficiency.

Cas9-mediated correction of the sickle mutation in human hematopoietic stem cells achieves curative levels of editing.

This study corrects the sickle mutation in human hematopoietic stem cells using a Cas9 RNP/ssDNA donor approach, showing long-term therapeutic editing in mice.