CRISPR gene editing – what are the possibilities for respiratory medicine?

Figures & data

Box 1. Adenine base editing (ABE) can be targeted to either strand [7]. Left – C > T mutation on top strand causes disease, ABE of A to G on bottom strand can potentially convert to WT. Right – G > A mutation on top strand causes disease, ABE of A to G on top strand can potentially convert to WT.

Box 2. The A > T mutation on top strand causes sickle cell disease (HBB^S). ABE of A to G on bottom strand cannot convert to HBB^WT, but can potentially convert to Makassar (HBB^G) and ameliorate disease severity by converting the Val codon to Ala.

Newby GA, Yen JS, Woodard KJ, et al. Base editing of haematopoietic stem cells rescues sickle cell disease in mice. Nature. 2021;595(7866):295–302. Available from: https://pubmed.ncbi.nlm.nih.gov/34079130/.

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