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Rational design of a compact CRISPR-Cas9 activator for AAV-mediated delivery

Vora S, Cheng J, Xiao R, VanDusen NJ, Quintino L, Pu WT, Vandenberghe LH, Chavez A, Church G
bioRxiv 2018. 298620 (Link opens in a new window) Article

Plasmids from Article

ID Plasmid Purpose
99654dSp-p65 Full (1-261)Expresses dSp Cas9 fused to p65 activation domain
99655dSp-p65 (150-261)Expresses dSp Cas9 fused to truncated p65 activation domain
99656dSp-p65 (100-261)Expresses dSp Cas9 fused to truncated p65 activation domain
99657dSp-p65 (200-261)Expresses dSp Cas9 fused to truncated p65 activation domain
99658dSp-p65 (1-200)Expresses dSp Cas9 fused to truncated p65 activation domain
99659dSp-p65 (1-150)Expresses dSp Cas9 fused to truncated p65 activation domain
99660dSp-p65 (1-100)Expresses dSp Cas9 fused to truncated p65 activation domain
99661dSp-p65 (50-150)Expresses dSp Cas9 fused to truncated p65 activation domain
99662dSp-Rta Full (1-190)Expresses dSp Cas9 fused to truncated RTA activation domain
99663dSp-Rta 75-190Expresses dSp Cas9 fused to truncated RTA activation domain
99664dSp-Rta 125-190Expresses dSp Cas9 fused to truncated RTA activation domain
99665dSp-Rta 50-175Expresses dSp Cas9 fused to truncated RTA activation domain
99666dSp-Rta 75-175Expresses dSp Cas9 fused to truncated RTA activation domain
99667dSp-Rta 100-175Expresses dSp Cas9 fused to truncated RTA activation domain
99668dSp-Rta 125-175Expresses dSp Cas9 fused to truncated RTA activation domain
99669dSp-VP64-RTA(75-190)Expresses dSp Cas9 fused to combination of truncated activation domains
99670dSp-VP64-p65-RTA(75-190)Expresses dSp Cas9 fused to combination of truncated activation domains
99672dSp-VP64-p65(100-261)-RTA(75-190)Expresses dSp Cas9 fused to combination of truncated activation domains
99673dSp-VP64-p65(100-261)-RTA(125-190)Expresses dSp Cas9 fused to combination of truncated activation domains
99674dSp-VP64-p65(150-261)-RTA(125-190)Expresses dSp Cas9 fused to combination of truncated activation domains
99675dSp-VP64-RTAExpresses dSp Cas9 fused at C term. To VP64 and RTA activation domains
99679pAAV-dSa-VP64-p65(100-261)-RTA(125-190)Expresses dSa Cas9 fused to optimized combination of truncated activation domains
99680pAAV-dSa-VP64Expresses dSa Cas9 fused to gold standard VP64 activator
99685pAAV-SCP1-dSa-VPR mini.Expresses dSa Cas9 fused to optimized combination of truncated activation domains from SCP1 promoter
99686pAAV-EFS-dSa-VPR mini.Expresses dSa Cas9 fused to optimized combination of truncated activation domains from EFS promoter
99687pAAV-CMV-dSa-VPR mini.-1X snRP1Expresses dSa Cas9 fused to optimized combination of truncated activation domains with 17 nt snRP1 poly adenylation signal
99688pAAV-CMV-dSa-VPR mini.-2X snRP1Expresses dSa Cas9 fused to optimized combination of truncated activation domains with 34 nt dual snRP1 poly adenylation signal
99689pAAV-CMV-dSa-VPR mini.-syn pAExpresses dSa Cas9 fused to optimized combination of truncated activation domains with syn pA (poly adenylation) signal
99690pAAV-SCP1-dSa VPR mini.-2X snRP-1 Actc1Expresses dSa Cas9 fused to optimized combination of truncated activation domains from SCP1 promoter with dual snRP1 poly adenylation signal, and Sa sgRNA for Actc1, vector allows for strong activation of mouse Actc1, can be packaged and delivered as AAV
99691pAAV-CMV-dSa VP64 Actc1Expresses dSa Cas9 fused to gold standard VP64 activator domain and Sa sgRNA for Actc1 promoter, vector allows for activation of mouse Actc1, can be packaged and delivered as AAV
99692pAAV-SCP1-dSa VPR mini.-2X snRP-1 HbbExpresses dSa Cas9 fused to optimized combination of truncated activation domains from SCP1 promoter with dual snRP1 poly adenylation signal, and Sa sgRNA for Hbb, vector allows for strong activation of mouse Hbb, can be packaged and delivered as AAV
99693pAAV-CMV-dSa VP64 HbbExpresses dSa Cas9 fused to gold standard VP64 activator domain and Sa sgRNA for Hbb promoter, vector allows for activation of mouse Hbb, can be packaged and delivered as AAV
99694pAAV-SCP1-dSa VPR mini.-2X snRP-1 Neurog2Expresses dSa Cas9 fused to optimized combination of truncated activation domains from SCP1 promoter with dual snRP1 poly adenylation signal, and Sa sgRNA for Actc1, vector allows for strong activation of mouse Actc1, can be packaged and delivered as AAV
99695pAAV-CMV-dSa VP64 Neurog2Expresses dSa Cas9 fused to gold standard VP64 activator domain and Sa sgRNA for Neurog2 promoter, vector allows for activation of mouse Neurog2, can be packaged and delivered as AAV

Antibodies from Article