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Multiplex epigenome editing of MECP2 to rescue Rett syndrome neurons.

Qian J, Guan X, Xie B, Xu C, Niu J, Tang X, Li CH, Colecraft HM, Jaenisch R, Liu XS
Sci Transl Med. 2023 Jan 18;15(679):eadd4666. doi: 10.1126/scitranslmed.add4666. Epub 2023 Jan 18. (Link opens in a new window) PubMed (Link opens in a new window) Article

Plasmids from Article

ID Plasmid Purpose
194885FUW-crRNA1and2-U6_TRE-dLbCpf1-NLS-hCTCF_WT-HA-P2A-tagBFPDox-inducible all-in-one lentiviral construct to express dCpf1-CTCF-WT and crRNA-1 and -2 targeting the human MECP2 locus.
194886FUW-crRNA1and2-U6_TRE-dLbCpf1-NLS-hCTCF_K365A_R368A-HA-P2A-tagBFPDox-inducible all-in-one lentiviral construct to express dCpf1-CTCF-K365A_R368A mutant and crRNA-1 and -2 targeting the human MECP2 locus.
194887FUW-crRNA1and2-U6_TRE-dLbCpf1-NLS-hCTCF_Q418A-HA-P2A-tagBFPDox-inducible all-in-one lentiviral construct to express dCpf1-CTCF-Q418A mutant and crRNA-1 and -2 targeting the human MECP2 locus.
194888FUW-crRNA1and2-U6_TRE-dLbCpf1-NLS-hCTCF_R396A-HA-P2A-tagBFPDox-inducible all-in-one lentiviral construct to express dCpf1-CTCF-R396A mutant and crRNA-1 and -2 targeting the human MECP2 locus.
194889FUW-crRNA-1-U6_TRE-dLbCpf1-NLS-hCTCF_WT-HA-P2A-tagBFPDox-inducible all-in-one lentiviral construct to express dCpf1-CTCF-WT and crRNA-1 targeting the human MECP2 locus.
194890FUW-crRNA-1-U6_TRE-dLbCpf1-NLS-hCTCF_K365A_R368A-HA-P2A-tagBFPDox-inducible all-in-one lentiviral construct to express dCpf1-CTCF-K365A_R368A mutant and crRNA-1 targeting the human MECP2 locus.
194891FUW-crRNA-1-U6_TRE-dLbCpf1-NLS-hCTCF_Q418A-HA-P2A-tagBFPDox-inducible all-in-one lentiviral construct to express dCpf1-CTCF-Q418A mutant and crRNA-1 targeting the human MECP2 locus.
194893FUW-crRNA-2-U6_TRE-dLbCpf1-NLS-hCTCF_WT-HA-P2A-tagBFPDox-inducible all-in-one lentiviral construct to express dCpf1-CTCF-WT and crRNA-2 targeting the human MECP2 locus.
194894FUW-crRNA-2-U6_TRE-dLbCpf1-NLS-hCTCF_K365A_R368A-HA-P2A-tagBFPDox-inducible all-in-one lentiviral construct to express dCpf1-CTCF-K365A_R368A mutant and crRNA-2 targeting the human MECP2 locus.
194895FUW-crRNA-2-U6_TRE-dLbCpf1-NLS-hCTCF_Q418A-HA-P2A-tagBFPDox-inducible all-in-one lentiviral construct to express dCpf1-CTCF-Q418A mutant and crRNA-2 targeting the human MECP2 locus.
194896FUW-crRNA-2-U6_TRE-dLbCpf1-NLS-hCTCF_R396A-HA-P2A-tagBFPDox-inducible all-in-one lentiviral construct to express dCpf1-CTCF-R396A mutant and crRNA-2 targeting the human MECP2 locus.
194897FUW-crRNA-scrambled-U6_TRE-dLbCpf1-NLS-hCTCF_WT-HA-P2A-tagBFPDox-inducible all-in-one lentiviral construct to express dCpf1-CTCF-WT and scrambled crRNA without target in human genome.
194898pgRNA_hMECP2_promoter_No3Lentiviral construct with mCherry and Puro cassettes to express sgRNA-3 targeting the promoter of human MECP2
194899pgRNA_hMECP2_promoter_No4Lentiviral construct with mCherry and Puro cassettes to express sgRNA-4 targeting the promoter of human MECP2
194900pgRNA_hMECP2_promoter_No6Lentiviral construct with mCherry and Puro cassettes to express sgRNA-6 targeting the promoter of human MECP2.
194901pgRNA_hMECP2_promoter_No7Lentiviral construct with mCherry and Puro cassettes to express sgRNA-7 targeting the promoter of human MECP2.
194902pgRNA_hMECP2_promoter_No8Lentiviral construct with mCherry and Puro cassettes to express sgRNA-8 targeting the promoter of human MECP2.
194903pgRNA_hMECP2_promoter_No9Lentiviral construct with mCherry and Puro cassettes to express sgRNA-9 targeting the promoter of human MECP2.

Antibodies from Article