CRISPR/Cas9-rAAV

Overview
CRISPR is a state-of-the-art genetic modification tool that can be used to knock out, mutate, or overexpress a gene of interest. CRISPR-based research strategies have been widely employed across biological fields of study due to their powerful performance and relatively straightforward mechanism of action.

CRISPR gene editing requires the exogenous expression of the Cas9 enzyme and a 15-20nt guide RNA targeted to the experiment’s gene of interest. Recent advances have made the design, construction, and use of the CRISPR systems increasingly convenient. PackGene’s pre-made Cas9 vectors build on this convenience by providing packaged AAV vectors for Cas9 expression at lightning-fast turnaround times.

PackGene provides pre-made “off the shelf” CRISPR-AAV vectors that drive Cas9 expression under the EFS, miniCMV, or CMV broad-spectrum promoters as well as several tissue-specific promoters in the AAV9 serotype. Our in-stock pre-made products are shipped within one business day, making these products ideal for projects with a constrained timeline and that require standardized vectors.

PackGene provides high quality packaged AAVs for CRISPR Cas9 or CRISPR Cpf1 expression that can be directly applied in vivo and include a complete quality inspection report.

Products
Promoter type Catalog No. Promoter and Gene

Universal promoter

AAV-XA20

ssAAV.miniCMV.SpCas9

AAV-XA02

ssAAV.EFS.SpCas9

AAV-XB02

ssAAV.EFS.SpCas9HF

AAV-XB06

ssAAV.miniCMV.SpCas9HF

AAV-AC010

ssAAV.EFS.SaCas9

AAV-AC040

ssAAV.CMV.SaCas9

AAV-AD040

ssAAV.CMV.SaCas9HF

AAV-AD010

ssAAV.EFS.SaCas9HF

AAV-XE01G

ssAAV.EFS.AsCpfl

AAV-XE01H

ssAAV.EFS.LbCpfl

AAV-XE02H

ssAAV.miniCMV.LbCpfl

Tissue-specific promotor

AAV-AC050

ssAAV.TBG.SaCas9

AAV-AC060

ssAAV.Elastasel.SaCas9

AAV-AC070

ssAAV.Rat Insulin 2.SaCas9

AAV-AC080

ssAAV.mouse Insulin2.SaCas9

AAV-AC090

ssAAV.hDesmin.SaCas9

AAV-AC100

ssAAV.hSynapsin.SaCas9

AAV-AC120

ssAAV.pMECP2.SaCas9

AAV-AC190

ssAAV.CBh.Sacas9.WPRE.SV40pA

AAV-AC071

ssAAV.Rat Insulin 2.SaCas9.U6.(Sa)sgRNA

AAV-AC081

ssAAV.mouse Insulin2.SaCas9.U6.(Sa)sgRNA

AAV-AC091

ssAAV.hDesmin.SaCas9.U6.(Sa)sgRNA

AAV-AC101

ssAAV.hSynapsin.SaCas9.U6.(Sa)sgRNA
AAV-AC121 ssAAV.pMECP2.SaCas9.U6.(Sa)sgRNA
AAV-AD060 ssAAV.Elastasel.SaCas9HF
AAV-AD070 ssAAV.rlnsulin2.SaCas9HF
AAV-AD080 ssAAV.mlnsulin2.SaCas9HF
AAV-AD090 ssAAV.hDesmin.SaCas9HF
AAV-AD100 ssAAV.hSynapsin.SaCas9HF
AAV-AD120 ssAAV.MECP2.SaCas9HF

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AAV
Case Study
Products: CRISPR AAV vectors from PackGene Biotech
Injection Method: Intramuscular Injection
Recommended Dose: 25 μL, 1.5E+12 vg/ml
Targeting Site: muscle
Animal Model: DMD mouse model
Journal: Genome Medicine, 2021 (IF=15.266)
Paper Title: In vivo genome editing in mouse restores dystrophin expression in Duchenne muscular dystrophy patient muscle fibers
DOI: 10.1186/s13073-021-00876-0
AAV production and injection
CRISPR AAV vectors were generated by PackGene Biotech Co. For intramuscular injection into NSI mice, the animals were anesthetized by abdominal injection of 5% chloral hydrate, followed by
injection of CRISPR AAV vector (25 μL, 1.5E+12 vg) into the TA muscle transplanted with DMD–MDSCs.
More Study Cases of PackGene’s AAV Services, please click here

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