Collectively, epitope engineering of hematopoietic stem cells represents a versatile platform to expand the therapeutic window of precision immunotherapies and may enable safer, more effective combinatorial treatment strategies for both nonmalignant and malignant hematologic conditions.
Joanne Baek, G. Casirati, Pietro Genovese et al.· Blood Advances· 0 citations
Epitope editing of KIT enables antibody-based, non-genotoxic conditioning that selectively enriches therapeutic BCL11A-edited haematopoietic stem/progenitor cells, supports durable engraftment, preserves clonal diversity and enhances induction of fetal haemoglobin, a therapeutic approach for conditions such as sickle c...
G. Casirati, Andrea Cosentino, Marta Freschi et al.· Nature· 0 citations
Efficient and precise correction of STAT1 GOF mutations by base editing is demonstrated, with maintenance of the correction through long-term engraftment in vivo, which represents the first application of gene editing to correct a dominant gain-of-function mutation causing immunodeficiency.
Robert Torrance, K. Orf, Nathan White et al.· Blood· 0 citations
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