CRISPR/Cas9 AAV Production Service

The CRISPR–Cas9 system enables programmable genetic and epigenetic manipulations which present exciting opportunities for personalized therapeutics. As CRISPR–Cas9 approaches the clinic, efficacy and safety for the patient are overriding. On that note, many have endeavored to deploy CRISPR–Cas9 with adeno-associated virus (AAV), delivery vectors that are prevalent, serologically compatible with a large fraction of the human population, and generally considered nonpathogenic. Besides, AAVs allow semiselective tissue tropism via local or systemic delivery.

The AAV-CRISPR system is particularly useful for editing disease-associated genes in the brain or central nervous system of mice due to the inability of most cationic nanocarriers to cross the blood-brain barrier, high transduction efficiency of AAV vectors in the brain, and non-dividing properties of neurons for long-term therapeutic effects. The AAV-CRISPR system has also been successfully used to restore the gene function in muscle-associated diseases such as Duchenne muscular dystrophy and diseases associated with the liver, heart, eye, and lung. This AAV-CRISPR-mediated gene editing in mice provided proof-of-principle studies for human disease modeling, gene therapy, or gene functional characterizations.

QVirus™ Platform has launched a comprehensive AAV packaging service combined with CRISPR/Cas9, the versatile genome-editing platform. Bringing together the versatile CRISPR/Cas9 genome editing system with powerful recombinant AAV (rAAV) technology, QVirusTM Platform’s AAV-Cas9 vectors extend genome editing capabilities to cutting edge in vivo applications. We are offering a Two-Vector as well as an All-In-One vector CRISPR-Cas9 System services:

AAV-Cas9 Two-vector System

For situations where you would like to introduce saCas9 and gRNA separately, QVirus™ Platform offers a two-vector AAV-Cas9 system service. The gRNA-expressing vector can also contain a fluorescent marker to verify transduction efficiency.

AAV-Cas9 All-In-One Vector

For the most streamlined in vivo introduction of Cas9 using rAAV technology, QVirus™ Platform offers an easy-to-use all-in-one AAV-Cas9 vector.

Features

  • Utilizing Staphylococcus aureus Cas9 (SaCas9)
  • Ensure the security of recombinant AAV
  • Broad range of host and specificity targeting
  • Stable physical properties

Application

  • Deliver Cas9 in vivo
  • Genome editing system of somatic tissues in postnatal animals.
  • Generate novel disease models
  • Develop gene therapies in small animal models

QVirus™ Platform has developed AAV-SaCas9 with different tropisms and serotypes to achieve in vivo gene knockout with high efficiency. If you have any special requirements, please feel free to contact us.

References
1. Lau C H , Suh Y . In vivo genome editing in animals using AAV-CRISPR system: Applications to translational research of human disease. F1000 Research, 2017, 6.
2. Chew W L, et al. A multifunctional AAV–CRISPR–Cas9 and its host response. Nature methods, 2016, 13(10): 868.

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