Sickle-cell anemia gene therapy

The emergence of highly efficient gene-editing technologies has shown promise for developing new therapeutic tools for human genetic disorders. Although past studies using CRISPR–Cas9 have

yielded encouraging results, by-products such as chromosomal mutations from Cas9-mediated double-strand breaks hinder clinical translation. Adenine base editors (ABEs) can efficiently

convert A–T base pairs to G–T pairs when guided to a site of interest by catalytically impaired CRISPR–Cas9, and were developed through directed evolution of a transfer-RNA adenosine

deaminase. Newby et al. have now demonstrated durable editing of the sickle-cell disease allele in the β-globin gene into a non-pathogenic variant in human-patient hematopoietic stem and

progenitor cells that were transplanted into mice. Editing successfully mediated phenotypic rescue, thus suggesting a potential one-time ABE treatment for sickle-cell disease.

Anyone you share the following link with will be able to read this content: