Base editing of trinucleotide repeats that cause Huntington’s disease and Friedreich’s ataxia reduces somatic repeat expansions in patient cells and in mice
Matuszek Z, Arbab Z, Kesavan M, Hsu A, Roy JCL, Zhao J, Yu T, Weisburd B, Newby GA, Doherty NJ, Wu M, Shibata S, Cristian A, Tao YA, Fearnley LG, Bahlo M, Rehm HL, Xie J, Gao G, Pinto RM, Liu DR
Nat Genet (2025)
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Article
Plasmids from Article
| ID | Plasmid | Purpose |
|---|---|---|
| 232720 | p2T-CMV-ABE8e_dNRCH-BlastR | A-to-G base editor |
| 232721 | p2T-CMV-EA-evoAPOBEC1max_NG-BlastR | C-to-T base editor |
| 232722 | p2T-CMV-EA-evoAPOBEC1max-32NLS_NG-BlastR | C-to-T base editor |
| 232723 | p2T-U6-sgCTG | SpCas9 guide RNA targeting CAG repeats for C-to-T base editing |
| 232724 | p2T-U6-sgGAA | Cas9 guide RNA targeting GAA repeats for A-to-G base editing |