Cat.No. : AAV00343Z
Titer: ≥1x10^12 GC/mL / ≥1x10^13 GC/mL Size: 30 ul/100 ul/500 ul/1 ml
Serotype: AAV Serotype 9 Storage: -80 ℃
| Cat. No. | AAV00343Z |
| Description | Premade AAV particles in serotype 9 express Streptococcus pyogenes Cas9 (SpCas9) from the EFS promoter. |
| Serotype | AAV Serotype 9 |
| Target Gene | SpCas9 |
| Titer | Varies lot by lot, typically ≥1x10^12 GC/mL |
| Size | Varies lot by lot, for example, 30 μL, 50 μL, 100 μL etc. |
| Storage | Store at -80℃. Avoid multiple freeze/thaw cycles. |
| Shipping | Frozen on dry ice |
| Creative Biogene ensures high-quality AAV particles by optimizing and standardizing production protocols and performing stringent quality control (QC). The specific QC experiments performed vary between AAV particle lots. | |
| Endotoxin | Endotoxins, primarily derived from Gram-negative bacteria, can trigger adverse immune responses. Endotoxin contamination is a significant concern in the production of AAV, especially for applications in animal studies and gene therapy. Effective endotoxin quality control is essential in the development and manufacturing of AAV particles. Creative Biogene utilizes rigorous endotoxin detection methods to monitor the endotoxin level in our produced AAV particles to ensure regulatory compliance. |
| Purity | AAV purity is critical for ensuring the safety and efficacy of AAV-based applications.AAV capsids are composed of three main protein components, known as viral proteins: VP1, VP2, and VP3. These proteins play a critical role in the structure and functionality of the AAV capsid. Monitoring the VP1, VP2, and VP3 content in AAV preparations is essential for quality control in AAV production. Our AAV particles are tested for showing three clear bands of VP1, VP2 VP3 by SDS-PAGE. |
| Sterility | The AAV virus samples are inoculated into the cell culture medium for about 5 days to detect bacterial and fungal growth. |
| Transducibility | Upon requirement, Creative Biogene can perform in vitro or in vivo transduction assays to evaluate the ability of AAV to deliver genetic material into target cells or tissues, and assess gene expression and functional activities. |
| Empty vs. Full Capsids | Based-on our proprietary AAV production and purification technology, Creative Biogene can always offer AAV particles with high ratio of full capsids. If required, we can also assess the ratio for a specifc lot of AAV particles by transmission electron microscopy (TEM) or other methods. |
Hemophilia is an incurable genetic disease. Although innovative treatments such as gene therapy or bispecific antibody therapies have been introduced, there remains a large unmet need in terms of achieving long-term therapeutic effects and treatment options for patients with inhibitors. Antithrombin (AT) is an endogenous negative regulator of thrombin generation and a potent genome editing target for the sustainable treatment of patients with hemophilia A and B. In this study, researchers developed and optimized lipid nanoparticles (LNPs) to deliver Cas9 mRNA as well as a single guide RNA targeting AT in mouse liver. LNP-mediated CRISPR-Cas9 delivery resulted in inhibition of AT, thereby improving thrombin generation. Both hemophilia A and B mice restored the bleeding-related phenotype. No active off-target, liver-induced toxicity, and significant anti-Cas9 immune response were detected, indicating that LNP-mediated CRISPR-Cas9 delivery is a safe and effective treatment for hemophilia.
A substantial safety concern of LNP-CRISPR is immune response or inflammation. Plasma was analyzed for aspartate aminotransferase (AST) and alanine transferase (ALT), and there were few differences between the groups. Therefore, LNP-CRISPR-mAT administration did not cause liver damage. In addition, the researchers used serum from LNP-injected mice to study systemic anti-Cas9 antibody responses by enzyme-linked immunosorbent assay (ELISA). Repeated LNP-CRISPR-mAT injections did not induce anti-Cas9 immunoglobulin G (IgG) compared to LNP empty treatment (Figure 1). However, when mice were injected intravenously with AAV9-EFS-SpCas9, systemic elevated anti-Cas9 IgG was detected after 6 weeks of treatment (Figure 1). This suggests that repeated LNP-CRISPR injections are a relatively less immunogenic method than continuous AAV-mediated Cas9 expression.
Figure 1. Serum anti-SpCas9 IgG concentrations after repeated injections of LNP-CRISPR-mAT. Mice injected intravenously with AAV9-EFS-SpCas9 (5X1013 vg/kg) were also tested 6 weeks after treatment. (Han J P, et al., 2022)
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