Mark C. Walters

Oligonucleotide synthesis errors are a source of untoward variation in HDR-mediated gene editing

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

Combinatorial base editing couples disease correction with lineage amplification in hematopoietic stem and progenitor cells

This paper develops a combinatorial base editing strategy to enhance erythroid lineage amplification and disease correction in hematopoietic stem and progenitor cells.

CRISPR-Cas9 interrogation of a putative fetal globin repressor in human erythroid cells.

This study uses CRISPR-Cas9 to test a putative fetal globin repressor region, finding it does not autonomously silence gamma-globin, suggesting it is not a therapeutic target for beta-hemoglobinopathies.

High-level correction of the sickle mutation amplified in vivo during erythroid differentiation

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

Efficient Correction of the Sickle Mutation in Human Hematopoietic Stem Cells Using a Cas9 Ribonucleoprotein Complex

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.