Researchers from Standford just published a paper in Nature featuring another promising application of CRISPR/Cas9, this time in sickle cell disease. After Bluebird’s somewhat disappointing LentiGlobin update from last week’s ASH abstract release, new approaches are welcome.
The Stanford researchers illustrate that ex vivo gene corrected haematopoietic stem cells followed by autologous transplantation can be used to effectively cure the genetic defect.
They use the CRISPR/Cas9 gene-editing system to create a homologous recombination at the HBB gene in haematopoietic stem cells. According to the paper, their enrichment model leads to a 90% targeted integration.Sounds promising.
According to a Reuters article, the team is eager to bring their approach to the clinic as soon as possible.