作者
Nipul Patel, Michael Pettiglio, Christopher Cummins, Jeff Pimentel, Anjali Kapuria, Azita Ghodssi, Meltem Isik, Alejandra Falla, Ravindra Amunugama, Jessica Lisle, Shu Wang, Michelle Lin, John Lydeard, Gary Ge, Dane Hazelbaker, Elizabeth Paik, Tirtha Chakraborty
发表日期
2021/11/23
期刊
Blood
卷号
138
页码范围
2939
出版商
Content Repository Only!
简介
Genome engineering of allogeneic hematopoietic stem cell transplants (HSCT), in which leukemia-specific cell surface antigens are removed by editing from the donor graft, may provide high therapeutic index and safety in combination with post-transplant targeted therapeutics. This strategy allows the therapeutics to specifically target leukemic cells by sparing the gene-edited graft, and thereby enable the next generation of HSCTs for treatment of acute myeloid leukemia (AML). So far, no single target antigen is known to be expressed in 100% of AML blasts, leukemic stem cells, or even in affected patients. Therefore, use of combinatorial therapies targeting multiple antigens may represent a potential paradigm shift in the standard of care for AML. Multiplex editing of HSCT in which two or more target antigens are removed from the same cell would enable combinatorial targeting while simultaneously protecting the …