Evaluation of persistence and fate of ex vivo edited-HSC modified with donor template and Its role in correcting Sickle Cell Disease
Sowmya Pattabhi, Samantha N. Lotti, Mason P. Berger, Swati Singh, David J. Rawlings
This study evaluates the efficacy of ssODN and rAAV6 donor templates in correcting sickle cell disease via ex vivo gene editing of HSCs, showing ssODN achieves higher in vivo gene correction.
bioRxiv · Jun 30, 2021 · PreprintGenomics