Re­searchers inch clos­er in ef­fec­tive­ly (and non-tox­i­cal­ly) mar­ry­ing CRISPR and CAR-T

CAR-T ther­a­pies tra­di­tion­al­ly have been made us­ing virus vec­tors, such as lentivirus or γ-retro­virus, to trans­fer the en­gi­neered can­cer-tar­get­ing re­cep­tor gene to a pa­tient’s T cells. But that process is com­pli­cat­ed and re­source-in­ten­sive, and the size of the vec­tors lim­its how much DNA they can car­ry.

At the same time CAR-T ther­a­pies were emerg­ing, CRISPR/Cas9 gene edit­ing was mak­ing its own break­throughs, and it pre­sent­ed it­self as a po­ten­tial al­ter­na­tive for edit­ing T cells. How­ev­er, com­bin­ing the two tech­nolo­gies proved chal­leng­ing on two fronts: For one, naked (not in a vec­tor) dou­ble-strand­ed DNA tem­plates are tox­ic; and two, CRISPR, while ef­fec­tive at mak­ing knock­out mu­ta­tions, was not very good at mak­ing knock-in mu­ta­tions, which are at the crux of CAR-T ther­a­py.

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