Article
Multiplex base editing to protect from CD33 directed drugs for immune and gene therapy.
Nature communications - 27 May 2025
Borot Florence, Humbert Olivier, Ehmsen Jeffrey T, Fields Emily, Kohli Sajeev, Radtke Stefan, Swing Kyle, Pande Dnyanada, Enstrom Mark R, Laszlo George S, Mayuranathan Thiyagaraj, Ali Abdullah Mahmood, Weiss Mitchell J, Yen Jonathan S, Newby Gregory A, Walter Roland B, Liu David R, Mukherjee Siddhartha, Kiem Hans-Peter
Abstract excerpt
The selection of genetically engineered immune or hematopoietic cells in vivo after gene editing remains a clinical problem and requires a method to spare on-target toxicity to normal cells. Here, we develop a base editing approach exploiting a naturally occurring CD33 single nucleotide polymorphism leading to removal of full-length CD33 surface expression on edited cells. CD33 editing in human and nonhuman...
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