PRJNA736715
Multiplex mutagenesis in human HSCs to enhance HbF reactivation
In the current study we explored for the first time, multiplex gene editing as a method to significantly enhance HbF reactivation, providing clinically relevant levels of engraftment and HbF expression after in vivo HSC gene editing. In vivo multiplex genome editing will potentially overcome several critical obstacles to a wide application of gene therapy for hemoglobinopathies, as the requirement for leukapheresis, ex vivo manipulation, repeat freezing/thawing of cells, myeloablation, and HSPC transplantation.