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Base-Edited CAR7 T Cells for Relapsed T-Cell Acute Lymphoblastic Leukemia

2023; Massachusetts Medical Society; Volume: 389; Issue: 10 Linguagem: Inglês

10.1056/nejmoa2300709

1533-4406

Robert Chiesa, Christos Georgiadis, Farhatullah Syed, Hong Zhan, Annie Etuk, Soragia Athina Gkazi, Roland Preece, Giorgio Ottaviano, Toni Braybrook, Jan Chu, Agnieszka Kubat, Stuart Adams, Rebecca Thomas, Kimberly Gilmour, David O’Connor, Ajay Vora, Waseem Qasim,

Virus-based gene therapy research

Cytidine deamination that is guided by clustered regularly interspaced short palindromic repeats (CRISPR) can mediate a highly precise conversion of one nucleotide into another — specifically, cytosine to thymine — without generating breaks in DNA. Thus, genes can be base-edited and rendered inactive without inducing translocations and other chromosomal aberrations. The use of this technique in patients with relapsed childhood T-cell leukemia is being investigated.