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J Immunol ; 207(3): 755-764, 2021 08 01.
Artículo en Inglés | MEDLINE | ID: mdl-34321286

RESUMEN

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 Ag receptor specificities, allowing for the generation of desirable Ab responses that cannot normally be elicited in animal models. Genome modification of B cells or their progenitor, hematopoietic stem cells, could potentially substitute Ab or protein replacement therapies that require multiple injections over the long term. To date, B cell editing using CRISPR-Cas9 has been solely employed in preclinical studies, in which 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.


Asunto(s)
Linfocitos B/fisiología , Terapia Genética/tendencias , Receptores de Antígenos de Linfocitos B/genética , Animales , Anticuerpos/genética , Anticuerpos/metabolismo , Sistemas CRISPR-Cas , Epítopos , Ingeniería Genética , Humanos , Inmunoterapia
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