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 M, 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, Mouro Pinto R, Liu DR
Nat Genet. 2025 May 26. doi: 10.1038/s41588-025-02172-8.
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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 |