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. PubMed Article
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. PubMed Article
Plasmids from Article
| ID | Plasmid | Purpose |
|---|---|---|
| 185910 | pCMV-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 |
| 185911 | pCMV-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 |
| 185912 | pCMV-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 |
| 185915 | pCMV-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 |
| 185916 | pCMV-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 |
| 185917 | pCMV-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 |
| 197499 | pCMV-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 |
| 197500 | pCMV-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 |
| 197501 | pCMV-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 |
| 197502 | pCMV-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 |
| 197503 | pCMV-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 |
| 197504 | pCMV-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 |
| 197505 | pCMV-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 |
| 197506 | pCMV-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 |
| 197507 | pCMV-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 |
| 197508 | pCMV-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 |
| 197509 | AAV-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) |
| 197510 | AAV-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) |
| 197511 | AAV-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 |