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Optimization of base editors for the functional correction of SMN2 as a treatment for spinal muscular atrophy.

Alves CRR, Ha LL, Yaworski R, Sutton ER, Lazzarotto CR, Christie KA, Reilly A, Beauvais A, Doll RM, de la Cruz D, Maguire CA, Swoboda KJ, Tsai SQ, Kothary R, Kleinstiver BP
Nat Biomed Eng. 2023 Dec 6. doi: 10.1038/s41551-023-01132-z. (Link opens in a new window) PubMed (Link opens in a new window) Article

Plasmids from Article

ID Plasmid Purpose
185910pCMV-T7-ABE8e-nSpCas9-P2A-EGFP (KAC978)pCMV and pT7 plasmid encoding human codon optimized ABE8e A-to-G base editor with nickase SpCas9(D10A) and P2A-EGFP
185911pCMV-T7-ABE8e-nSpG-P2A-EGFP (KAC984)pCMV and pT7 plasmid encoding human codon optimized ABE8e A-to-G base editor with nickase SpG(D10A/D1135L/S1136W/G1218K/E1219Q/R1335Q/T1337R) and P2A-EGFP
185912pCMV-T7-ABE8e-nSpRY-P2A-EGFP (KAC1069)pCMV and pT7 plasmid encoding human codon optimized ABE8e A-to-G base editor with nicakse SpRY(D10A/A61R/L1111R/D1135L/S1136W/G1218K/E1219Q/N1317R/A1322R/R1333P/R1335Q/T1337R) and P2A-EGFP
185915pCMV-T7-ABE8.20m-nSpCas9-P2A-EGFP (KAC1158)pCMV and pT7 plasmid encoding human codon optimized ABE8.20m A-to-G base editor with nickase SpCas9(D10A) and P2A-EGFP
185916pCMV-T7-ABE8.20m-nSpG-P2A-EGFP (KAC1160)pCMV and pT7 plasmid encoding human codon optimized ABE8.20m A-to-G base editor with nickase SpG(D10A/D1135L/S1136W/G1218K/E1219Q/R1335Q/T1337R) and P2A-EGFP
185917pCMV-T7-ABE8.20m-nSpRY-P2A-EGFP (KAC1335)pCMV and pT7 plasmid encoding human codon optimized ABE8.20m A-to-G base editor with nicakse SpRY(D10A/A61R/L1111R/D1135L/S1136W/G1218K/E1219Q/N1317R/A1322R/R1333P/R1335Q/T1337R) and P2A-EGFP
197499pCMV-T7-ABE8.20m-SpCas9-NRRH-P2A-EGFP (RMD47)CMV and T7 promoter expression plasmid for human codon optimized ABE(8.20) A-to-G base editor with nSpCas9-NRRH(D10A) and P2A-EGFP
197500pCMV-T7-ABE8.20m-SpCas9-NRTH-P2A-EGFP (RMD63)CMV and T7 promoter expression plasmid for human codon optimized ABE(8.20) A-to-G base editor with nSpCas9-NRTH(D10A) and P2A-EGFP
197501pCMV-T7-ABE8.20m-SpCas9-NRCH-P2A-EGFP (RMD55)CMV and T7 promoter expression plasmid for human codon optimized ABE(8.20) A-to-G base editor with nSpCas9-NRCH(D10A) and P2A-EGFP
197502pCMV-T7-ABE8e-SpCas9-NRRH-P2A-EGFP (RMD51)CMV and T7 promoter expression plasmid for human codon optimized ABE8e A-to-G base editor with nSpCas9-NRRH(D10A) and P2A-EGFP
197503pCMV-T7-ABE8e-SpCas9-NRTH-P2A-EGFP (RMD21)CMV and T7 promoter expression plasmid for human codon optimized ABE8e A-to-G base editor with nSpCas9-NRTH(D10A) and P2A-EGFP
197504pCMV-T7-ABE8e-SpCas9-NRCH-P2A-EGFP (RMD57)CMV and T7 promoter expression plasmid for human codon optimized ABE8e A-to-G base editor with nSpCas9-NRCH(D10A) and P2A-EGFP
197505pCMV-T7-ABE8e-SpCas9-HF1-P2A-EGFP (LM422)CMV and T7 promoter expression plasmid for human codon optimized ABE8e A-to-G base editor with nSpCas9-HF1(D10A) and P2A-EGFP
197506pCMV-T7-ABE8e-SpCas9-HiFi-P2A-EGFP (LM446)CMV and T7 promoter expression plasmid for human codon optimized ABE8e A-to-G base editor with nSpCas9-HiFi(D10A) and P2A-EGFP
197507pCMV-T7-ABE8e-SpRY-HF1-P2A-EGFP (LM434)CMV and T7 promoter expression plasmid for human codon optimized ABE8e A-to-G base editor with nSpRY-HF1(D10A) and P2A-EGFP
197508pCMV-T7-ABE8e-SpRY-HiFi-P2A-EGFP (LM458)CMV and T7 promoter expression plasmid for human codon optimized ABE8e A-to-G base editor with nSpRY-HiFi(D10A) and P2A-EGFP
197509AAV-pCBh-ABE8e-nSpCas9(D10A)[N-term] (HES1468)Cbh promoter expression plasmid for N-terminal intein-split AAV construct with N-term of ABE8e-nSpCas9(D10A)
197510AAV-pCbh-ABE8e-nSpCas9(D10A/A61R)[N-term] (CA19)Cbh promoter expression plasmid for N-terminal intein-split AAV construct with N-term of ABE8e-nSpCas9(D10A/A61R) (to be used with SpRY C-term constructs)
197511AAV-pCbh-SpRY[C-term]-gRNAentry (CA20)Cbh promoter expression plasmid for C-terminal intein-split AAV construct with C-term of SpRY and BsmBI entry cassette to clone SpCas9 gRNA spacer

Antibodies from Article