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Treatment of a severe vascular disease using a bespoke CRISPR-Cas9 base editor in mice.

Alves CRR, Das S, Krishnan V, Ha LL, Fox LR, Stutzman HE, Shamber CE, Kalailingam P, McCarthy S, Lino Cardenas CL, Fong CE, Imai T, Mitra S, Yun S, Wood RK, Benning FMC, Roh K, Lawton J, Kim N, Silverstein RA, Ferreira da Silva J, de la Cruz D, Richa R, Xie J, Gray-Edwards HL, Malhotra R, Chung DY, Chao LH, Tsai SQ, Maguire CA, Lindsay ME, Kleinstiver BP, Musolino PL
Nat Biomed Eng. 2025 Sep 11:10.1038/s41551-025-01499-1. doi: 10.1038/s41551-025-01499-1. (Link opens in a new window) PubMed (Link opens in a new window) Article

Plasmids from Article

ID Plasmid Purpose
242651pCMV-ABE8.8m-WT-P2A-EGFP (BKS953)CMV promoter expression plasmid for human codon optimized ABE8.8m A-to-G base editor with SpCas9(D10A) and P2A-EGFP
242652pCMV-ABE8.8m-SpG-P2A-EGFP (BKS965)CMV promoter expression plasmid for human codon optimized ABE8.8m A-to-G base editor with SpG(D10A) and P2A-EGFP
242653pCMV-ABE8.8m-VRQR-P2A-EGFP (BKS971)CMV promoter expression plasmid for human codon optimized ABE8.8m A-to-G base editor with nVRQR(D10A) and P2A-EGFP
242654pCMV-ABEmax-eVRQR-P2A-EGFP (LLH575)CMV promoter expression plasmid for human codon optimized ABEmax(7.10) A-to-G base editor with neVRQR(D10A) and P2A-EGFP
242655pCMV-ABE8.8m-eVRQR-P2A-EGFP (BKS974)CMV promoter expression plasmid for human codon optimized ABE8.8m A-to-G base editor with neVRQR(D10A) and P2A-EGFP
242656pCMV-ABE8.20m-eVRQR-P2A-EGFP (LLH569)CMV promoter expression plasmid for human codon optimized ABE8.20 A-to-G base editor with neVRQR(D10A) and P2A-EGFP
242657pCMV-ABE8e-eVRQR-P2A-EGFP (HES1425)CMV promoter expression plasmid for human codon optimized ABE8e A-to-G base editor with neVRQR(D10A) and P2A-EGFP
242658pRhaB-8xHis-ABE8e-eVRQR (LLH551)pRhaB promoter expression plasmid for ABE8e-eVRQR with an N-terminal His8-tag
242659N-term AAV-ABE8e-SpCas9(S55R) (CA21)CBh promoter expression plasmid for N-terminal intein-split AAV construct with N-term of ABE8e-SpCas9(S55R) - to be used with eVRQR constructs
242660C-term AAV-SpCas9-VRQR-gRNA_entry (HES1477)CBh promoter expression plasmid for C-terminal intein-split AAV construct with C-term of SpCas9-VRQR and pU6 SpCas9 gRNA entry cassette
242661C-term AAV-eVRQR-ACTA2-gRNA-A4 (human) (LLH301)CBh promoter expression plasmid for C-terminal intein-split AAV construct with C-term of SpCas9-VRQR and human gRNA-A4
242662C-term AAV-eVRQR-ACTA2-gRNA-A4 (mouse) (LLH310)CBh promoter expression plasmid for C-terminal intein-split AAV construct with C-term of SpCas9-VRQR and mouse gRNA-A4
242663pCMV-eVRQR-P2A-EGFP (KAC1028)CMV promoter expression plasmid for human codon optimized SpCas9-eVRQR(S55R)-BPNLS(SV40)-3xFLAG-P2A-EGFP
242664pCMV-SpCas9-VQR-P2A-EGFP (LRF199)CMV promoter expression plasmid for human codon optimized SpCas9-VQR-BPNLS(SV40)-3xFLAG-P2A-EGFP
242665pCMV-SpCas9-VRQR(+A61R)-P2A-EGFP (KAC1030)CMV promoter expression plasmid for human codon optimized SpCas9-VRQR(A61R)-BPNLS(SV40)-3xFLAG-P2A-EGFP
242666pCMV-SpCas9-VRQR(+R221K)-P2A-EGFP (LRF95)CMV promoter expression plasmid for human codon optimized SpCas9-VRQR(R221K)-BPNLS(SV40)-3xFLAG-P2A-EGFP
242667pCMV-SpCas9-VRQR(+L244Q)-P2A-EGFP (LRF96)CMV promoter expression plasmid for human codon optimized SpCas9-VRQR(L244Q)-BPNLS(SV40)-3xFLAG-P2A-EGFP
242668pCMV-SpCas9-VRQR(+D269A)-P2A-EGFP (LRF64)CMV promoter expression plasmid for human codon optimized SpCas9-VRQR(D269A)-BPNLS(SV40)-3xFLAG-P2A-EGFP
242669pCMV-SpCas9-VRQR(+G366R)-P2A-EGFP (KAC1026)CMV promoter expression plasmid for human codon optimized SpCas9-VRQR(G366R)-BPNLS(SV40)-3xFLAG-P2A-EGFP
242670pCMV-SpCas9-VRQR(+N394K)-P2A-EGFP (KAC956)CMV promoter expression plasmid for human codon optimized SpCas9-VRQR(N394K)-BPNLS(SV40)-3xFLAG-P2A-EGFP
242671pCMV-SpCas9-VRQR(+G1104K)-P2A-EGFP (LRF54)CMV promoter expression plasmid for human codon optimized SpCas9-VRQR(G1140K)-BPNLS(SV40)-3xFLAG-P2A-EGFP
242672pCMV-SpCas9-VRQR(+L1111H)-P2A-EGFP (LRF269)CMV promoter expression plasmid for human codon optimized SpCas9-VRQR(L1111H)-BPNLS(SV40)-3xFLAG-P2A-EGFP
242674pCMV-SpCas9-VRQR(+T1138K)-P2A-EGFP (LRF243)CMV promoter expression plasmid for human codon optimized SpCas9-VRQR(T1138K)-BPNLS(SV40)-3xFLAG-P2A-EGFP
242675pCMV-SpCas9-VRQR(+L1245V)-P2A-EGFP (LRF49)CMV promoter expression plasmid for human codon optimized SpCas9-VRQR(L1245V)-BPNLS(SV40)-3xFLAG-P2A-EGFP
242676pCMV-SpCas9-VRQR(+A1285K)-P2A-EGFP (KAC1134)CMV promoter expression plasmid for human codon optimized SpCas9-VRQR(A1285K)-BPNLS(SV40)-3xFLAG-P2A-EGFP
242677pCMV-SpCas9-VRQR(+N1286Q)-P2A-EGFP (LRF247)CMV promoter expression plasmid for human codon optimized SpCas9-VRQR(N1286Q)-BPNLS(SV40)-3xFLAG-P2A-EGFP
242678pCMV-SpCas9-VRQR(+N1317R)-P2A-EGFP (LRF53)CMV promoter expression plasmid for human codon optimized SpCas9-VRQR(N1317R)-BPNLS(SV40)-3xFLAG-P2A-EGFP
242680pCMV-SpCas9-VRQR(+D1332K)-P2A-EGFP (KAC966)CMV promoter expression plasmid for human codon optimized SpCas9-VRQR(D1332K)-BPNLS(SV40)-3xFLAG-P2A-EGFP
242681pCMV-SpCas9-VRQR(+D1332R)-P2A-EGFP (LRF45)CMV promoter expression plasmid for human codon optimized SpCas9-VRQR(D1332R)-BPNLS(SV40)-3xFLAG-P2A-EGFP
242682pCMV-pLenti-ACTA2-R179 (WT) (LLH661)Plasmid for expression of ACTA2-R179 (wild-type) cDNA from a lentiviral cassette
242683pCMV-pLenti-ACTA2-R179H (LLH647)Plasmid for expression of ACTA2-R179H cDNA from a lentiviral cassette
242684pCMV-pLenti-ACTA2-M178V (LLH709)Plasmid for expression of ACTA2-M178V cDNA from a lentiviral cassette
242685pCMV-pLenti-ACTA2-L180V (LLH669)Plasmid for expression of ACTA2-L180V cDNA from a lentiviral cassette
242686pCMV-pLenti-ACTA2-L180M (LLH673)Plasmid for expression of ACTA2-L180M cDNA from a lentiviral cassette
242687pCMV-pLenti-ACTA2-D181G (LLH681)Plasmid for expression of ACTA2-D181G cDNA from a lentiviral cassette
242688pCMV-pLenti-ACTA2-L180L (LLH677)Plasmid for expression of ACTA2-L180L cDNA from a lentiviral cassette
242689pCMV-PE2-SpG (HES824)Prime editing in mammalian cells with SpG-based PE2 prime editors

Antibodies from Article