AAV Capsid Discovery Kits

Key Benefits

Proven Performance Across In Vitro, Ex Vivo, and In Vivo Models

• Our AAV Capsid Screening Kits consistently deliver accurate performance across various models, including in vitro (cell lines, primary cells, iPSCs), ex vivo (organoids), and in vivo (mouse, xenograft, NHP) systems. This is supported by rigorous quality control that ensures consistent and dependable data, enabling confident AAV selection for all downstream applications.

  

  Westhaus et al. Human Gene Therapy. 2020. Drouyer et al. Molecular Therapy: Nucleic Acids. 2024.

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Frequently Asked Questions

• Q: What is the difference between the Generation 1 and Generation 2 Universal Panels?

  A: The Universal Panel (Generation 1) includes approximately 70 pre-validated serotypes, featuring 34 novel CMRI capsids, 12 PackGene capsids, and 24 published capsids. The Generation 2 panel expands this panel to approximately 99 serotypes by including 53 published capsids alongside the novel options.

• Q: How much do the screening kits cost?

  A: Pricing starts at approximately $6,000 for the Generation 1 Universal Panel and approximately $8,700 for the Generation 2 panel.

• Q: Can I add my own custom capsids to the screening pool?

  A: Yes. We offer Custom Add-Ons that allow you to include your own proprietary custom capsids into the screening panel alongside our pre-validated options.

• Q: What tissue-specific panels are available?

  A: You can augment your universal panel with capsids pre-selected for specific target tissues. Current Add-On options include CNS/Brain (21 serotypes), Eye/Retina (9 serotypes), Liver/Hepatocytes (5 serotypes), and Muscle & Heart (5 serotypes), with additional options available for Kidney, Lung, and T-Cells.

• Q: What experimental models are compatible with these kits?

  A: The single viral pool can be applied to a variety of models, including in vitro cultured cells, ex vivo organoids, and in vivo animal models.

• Q: How does the barcoding system work for the dual readout?

  A: Each capsid is packaged with a standardized EGFP reporter gene and a unique molecular barcode located in the 3′ UTR. To ensure rigorous internal quality control, the kits utilize dual 6-nt barcodes per capsid as technical duplicates. These barcodes are sequenced from extracted DNA to quantify viral entry and from extracted RNA (cDNA) to quantify functional transgene expression.

• Q: Is the kit compatible with single-cell RNA sequencing?

  A: At this time, the current version of the AAV Capsid Screening Kit is not compatible with 10X Genomics single-cell RNA-seq platforms.

• Q: How do you guarantee the uniformity and quality of the pooled library?

  A: Our libraries undergo rigorous NGS validation to ensure unbiased composition. The pool achieves elite Skew Ratios that far exceeding the Broad Institute’s standard for acceptable screening (<10). Furthermore, 100% of variants are detected with >22,000 reads, and Q30 scores >95% confirm the integrity of the barcode sequences.

• Q: Are the capsids in the pool perfectly equimolar, and do I need to normalize my data?

  A: The capsids are individually produced, pre-quantified, and pooled at close to equimolar ratios (achieving an elite Skew Ratio <4). While the library is delivered pre-pooled to save you from complex upfront titering, your final NGS data should still be normalized. By sequencing the residual input aliquot saved on the day of your experiment, you can accurately normalize your tissue output data against the exact starting frequencies of each capsid, ensuring precise enrichment and depletion metrics.

• Q: What are the recommended storage conditions for the kits?

  A: We strongly recommend storing the pre-normalized screening kits at 4°C, where they maintain optimal stability and performance for up to a year.

• Q: Can I store the AAV Capsid Screening Kit at -80°C?

  A: While many labs store AAV vectors at -80°C, freezing the pooled screening kit is generally discouraged due to the risk of differential damage and induced variability across the highly multiplexed kit. If long-term freezing at -80°C is absolutely necessary for your workflow, you must aliquot the pool upon initial receipt to strictly avoid any freeze-thaw cycles, which will disproportionately affect different serotypes and compromise the elite equimolarity of the screen.

• Q: Should I save a residual aliquot of the viral pool from my experiment?

  A: Yes, it is highly recommended that you save a residual aliquot of the kit at the time of your experiment. This residual serves as your baseline "input" control for downstream Next-Generation Sequencing (NGS) analysis. By comparing the barcode frequencies in your transduced tissue (the "output") against this specific residual aliquot, you can accurately calculate the true fold-change (enrichment or depletion) of each capsid variant while accounting for any minor sequencing biases or handling variations.