Vector Strategies to Actualize B Cell-Based Gene Therapies.
Vector Strategies to Actualize B Cell-Based Gene Therapies.
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DOI：
10.4049/jimmunol.2100340
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发表时间：
2021-08-01
期刊：
影响因子：
--
通讯作者：
Voss JE
中科院分区：
文献类型：
--
作者：
Jeske AM;Boucher P;Curiel DT;Voss JE
Recent developments in genome editing and delivery systems have opened new possibilities for B cell gene therapy. CRISPR/Cas9 nucleases have been used to introduce transgenes into B cell genomes for subsequent secretion of exogenous therapeutic proteins from plasma cells, and to program novel B cell antigen-receptor specificities, allowing for the generation of desirable antibody responses that cannot normally be elicited in animal models. Genome modification of B cells or their progenitor, hematopoietic stem cells (HSCs), could potentially substitute antibody or protein replacement therapies that often require multiple lifelong injections. To date, B cell editing utilizing CRISPR/Cas9 has been solely employed in preclinical studies wherein cells are edited ex vivo. In this review, we discuss current B cell engineering efforts and strategies for the eventual safe and economical adoption of modified B cells into the clinic, including in vivo viral delivery of editing reagents to B cells.
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