AAV Vector/transgene/Biomarker Protein Biodistribution Analysis

Gene therapy development requires a comprehensive understanding of the unique aspects of these products in relation to their in vivo safety profiles. And AAV-derived vectors are the most promising gene therapy tools, as their replication incompetence and ability to mediate long-term transgene expression. Biodistribution and viral shedding assays are two critical ways by which such safety can be assessed.

While qPCR-based methods provide average values for copy number, these provide only an average value for an entire complex tissue and lack cellular resolution. RNA in situ hybridization (ISH) technology provides morphology-based, cell-specific quantification of vector DNA, transgene mRNA, and specific biomarker protein. RNA ISH and IHC allow analysis of vector DNA, transgene mRNA, and biomarker protein, respectively, while preserving the cellular relationship and tissue architecture. Researchers have found that combining or multiplexing both technologies provides unique information. AAV Vector/transgene/Biomarker Protein Biodistribution Analysis service from Creative Bioarray can help you validate and accelerate your gene therapy development.

Features of AAV Vector/transgene/Biomarker Protein Biodistribution Analysis (ISH + IHC method)

(1) Detection of single-copy DNA vectors
(2) Codon-optimized transgene analysis
(3) Biomarker protein analysis
(4) Custom probes designed within 1-2 weeks
(5) Fastest turnaround time
(6) Experienced scientists for accurate data analysis
(7) Standard and customized experiment plan

Benefits of AAV Vector/Transgene/Biomarker Protein Biodistribution Analysis (ISH + IHC method)

(1) Simultaneously visualize in vivo delivery of vector DNA and therapeutic transgene mRNA
(2) Identify cell tropism of AAV vector by multiplexing with specific biomarker protein
(3) Distinguish transgene from endogenous sequences, at the single nucleotide level
(4) Quantify AAV+ cell number and track persistence over time

Creative Bioarray offers AAV Vector/Transgene/Biomarker Protein Biodistribution Analysis for you as follows:

• Probe design
• Probe synthesis
• ISH + IHC staining
• Imaging
• Data analysis

Quotation and ordering

Our customer service representatives are available 24hr a day! We thank you for considering Creative Bioarray as your AAV Vector/Transgene/Biomarker Protein Biodistribution Analysis partner.

References

1. Hastie E and Samulski RJ. Adeno-associated virus at 50: A golden anniversary of discovery, research, and gene therapy success — a personal perspective. Hum Gene Ther. 2015; 26:257–265
2. Wang F, et al. RNAscope®: A novel in situ RNA analysis platform for formalin-fixed, paraffinembedded tissues. J Mol Diagn. 2012; 14(1):22–9.
3. Polinski NK, et al. Impact of age and vector construct on striatal and nigral transgene expression. Mol Ther Methods Clin Dev. 2016; 3, 16082
4. Keeler AM, et al. Cellular analysis of silencing the Huntington's Disease gene using AAV9 mediated delivery of artificial microRNA into the striatum of Q140/Q140 mice. J Huntingtons Dis. 2016; 5(3):239–248
5. Borel F, et al. Therapeutic rAAVrh10 mediated SOD1 silencing in adult SOD1G93A mice and nonhuman primates. Hum Gene Ther. 2016; 27(1):19–31.