CRISPR/Cas9 System Tools

CRISPR genome editing is a common system for in vitro and germline genome editing, but in vivo gene editing methods are limited due to Cas9 delivery challenges. Adeno-associated viral vectors (AAV) are effective tools for in vivo gene delivery with the advantages of low immunogenicity and the ability to infect specific tissues. Creative Biolabs' AAV CRISPR/Cas9 system vectors are perfect tools for CRISPR-based in vivo applications. They have increased specificity, an incredibly low off-target rate, higher stability, and greater adaptability.

The CRISPR-Cas9 system is an efficient gene editing technology, while AAV is an effective gene delivery vector. The combination of the two becomes a double-weapon for gene editing, which not only enhances precision and efficiency but also opens up new possibilities for gene therapy. Researchers utilize this technology to investigate gene function, disease models, and drug screening. In terms of practical applications, the combination of CRISPR-Cas9 and AAV offers fresh promise for treating genetic disorders. In the realm of hereditary blindness, researchers have employed CRISPR-Cas9 and AAV to successfully correct mutations in several blinding genes, restoring patients' vision.

CRISPR-Cas9 Gene Editing Service Workflow

Creative Biolabs has launched a one-stop CRISPR-Cas9 viral vector design super service.

Advantages of the CRISPR/Cas9 System Tools

• High efficiency: Our products edit genomes with precision and efficiency.
• Broad spectrum: No gene, cell or species restrictions.
• Multiple editing capabilities: Allows simultaneous gene targeting of multiple target sites.
• Multi-functional: It can achieve knock-out, knock-in, inhibition, and activation of target genes.

Case Study

Intravitreal injection of CjCas9 encoding AAV targeting Hif1a into mice resulted in the depletion of the Vegfa or Hif1a gene in the mouse retina within 6 weeks, as well as the effective inhibition of pathological choroidal neovascularization. These long-term observations can support in vivo editing of selected target genes linked with pathological conditions in a variety of retinal diseases, including age-related macular degeneration (AMD), Leber congenital amaurosis, and retinitis pigmentosa.

Fig.1 Histological staining evaluation of mouse retinal tissue at 14 months after intravitreal injection of AAV-CjCas9. A-B) Representative H&E images of mouse retinas 14 months after intravitreal injection of AAV encoding CjCas9, and the number of apoptotic cells. C) Representative immunofluorescence images of mouse retinas 14 months after intravitreal injection of AAV-encoded CjCas9.¹

To make it easy for you to realize your research goals and deliver high quality viral vectors, Creative Biolabs has launched a viral vector design super service. Contact us for more information and achieve AAV CRISPR/Cas9's tremendous potential for biological research and therapeutics.

Reference

1. Jo, Dong Hyun, et al. "Long-term effects of in vivo genome editing in the mouse retina using Campylobacter jejuni Cas9 expressed via adeno-associated virus." Molecular Therapy 27.1 (2019): 130-136.