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Targeted Transgene Activation in the Brain Tissue by Systemic Delivery of Engineered AAV1 Expressing CRISPRa.

Lau CH, Ho JW, Lo PK, Tin C
Mol Ther Nucleic Acids. 2019 Jun 7;16:637-649. doi: 10.1016/j.omtn.2019.04.015. Epub 2019 Apr 23. (Link opens in a new window) PubMed (Link opens in a new window) Article

Plasmids from Article

ID Plasmid Purpose
158972pAAV-pMecp2-dSaCas9-VP64-pU6-sgRNAVector C encodes pAAV-pMecp2-dSaCas9-VP64-spA-pU6-sgRNA (BsaI) transgenes for AAV packaging and expression of CRISPR activator in neurons
158987pAAV-pMecp2-dSaCas9-VP160-pU6-sgRNAVector D encodes pAAV-pMecp2-dSaCas9-VP160-spA-pU6-sgRNA (BsaI) transgenes for AAV packaging and expression of CRISPR activator in neurons
158988pAAV-pMecp2-dSaCas9-KRAB-pU6-sgRNAVector E encodes pAAV-pMecp2-dSaCas9-KRAB-spA-pU6-sgRNA (BsaI) transgenes for AAV packaging and expression of CRISPR interference in neurons
158989pAAV-pMecp2-dSaCas9-SID4X-pU6-sgRNAVector F encodes pAAV-pMecp2-dSaCas9-SID4X-spA-pU6-sgRNA (BsaI) transgenes for AAV packaging and expression of CRISPR interference in neurons
158990pAAV-pCMV-dSaCas9-VP64-pU6-sgRNAVector G encodes pAAV-pCMV-dSaCas9-VP64-spA-pU6-sgRNA (BsaI) transgenes for AAV packaging and expression of CRISPR activator in mammalian cells
159173pAAV-pMecp2-SaCas9-pU6-sgRNAVector A encodes pAAV-pMecp2-SaCas9-spA-pU6-sgRNA (BsaI) transgenes for AAV packaging and expression of SaCas9-sgRNA in neurons
159174pAAV-pMecp2-dSaCas9-pU6-sgRNAVector B encodes pAAV-pMecp2-dSaCas9-spA-pU6-sgRNA (BsaI) transgenes for AAV packaging and expression of dSaCas9-sgRNA in neurons

Antibodies from Article