Gregory A. Newby

This study corrects a CFTR splice site variant in airway cells using base editors and nanoparticles, showing increased CFTR expression and functional rescue in multiple cell types.

The paper uses CRISPR editing and multimodal single-cell sequencing to identify the functional effects of disease variants in primary human cells.

The paper uses base editor screens to assess phosphorylation site functionality in T cell activation, identifying novel sites that modulate transcriptional activity and signaling pathways.

This study demonstrates the use of protecting oligos to enhance crRNA stability and potency for in vivo genome editing via AAV co-delivery in mice.

This paper demonstrates nonviral base editing of a KCNJ13 mutation in a mouse model of retinal degeneration, preserving vision through functional rescue of Kir7.1 channels.

The paper evaluates CRISPR base editors for correcting a KCNJ13 mutation causing LCA16, finding high on-target efficiency but negligible channel function due to bystander edits and mRNA structural issues.

The paper identifies causal genetic variants in autoimmune diseases by combining allele-specific reporter assays with chromatin accessibility data, showing their impact on gene expression and T cell function.

ONE-seq identifies population-specific off-target effects of CRISPR gene editors using global human genome sequence diversity.

This paper compares CRISPR-mediated HDR and PE2 systems in mice to show PE2's higher fidelity in editing a TFBS, leading to more effective tissue-specific gene inactivation.