AAV Biodistribution & Shedding Assay Service

As one of the most actively investigated gene therapy vehicles, adeno-associated viruses (AAVs) have been used in hundreds of clinical trials worldwide, in part because AAVs are the safest and most effective gene delivery vehicles to achieve long-term transgene expression in gene therapy.

Whereas promising results have been obtained from clinical trials in the treatment of a number of diseases, the biodistribution of some AAV serotypes, especially those new discovered serotypes, has not been fully characterized. However, safety concerns have been raised for some AAV vectors administered in animal species and in humans. Therefore, it is important to determine the AAV biodistribution and shedding to assess the safety of AAV vectors prior to use of them in clinical trials.

The biodistribution assay relates to the spreading or localization of the AAV vector from the site of administration in the animal model, whereas the shedding assay is to investigate the dissemination of the AAV vector through secretions and/or excreta from the animal model. Different AAV serotypes, even the same serotype with different transgenes, may have different biodistribution and shedding. Currently, most biodistribution and shedding assays typically focus on intravenous injection without studying other routes of administration such as intramuscular, intra-arterial and intracranial.

Figure 1. Intrathecal administration of AAV-PHP.B leads to superior transduction in the Rhesus Macaque Cortex and Spinal Cord compared with intra-carotid administration. (Liguore WA etc. Mol Ther. 2019)

Creative Biogene owns highly experienced scientists along with our state-of-the-art facilities which enable us to provide full support to your AAV projects whether you are at early research stage, preclinical or clinical stage. We specialize in every aspect of AAV studies, covering AAV vector design, virus production, capsid evolution, as well as biodistribution and shedding analysis.

Highlights

Our one-stop AAV platform allows us to support your research/preclinical/clinical needs.

We can use multiple preclinical species for characterizing AAV biodistribution and shedding which includes mouse, rat as well as nonhuman primate (NHP).

A wide range of methods have been developed by our scientists for use in determining AAV biodistribution at the organ, tissue and cell level. These methods include fluorescence imaging, flow cytometry, qPCR, RT-PCR, ddPCR, western blot, ELISA etc.

We are capable of producing AAV virus particles in a variety of serotypes including AAV1-9, DJ, DJ/8, PHP.B, PHP.eB, PHP.S, rh10, retrograde, AAV2.7m8, BR1 etc.

Our highly trained experts can perform multiple routes of AAV administration including intramuscular, intravenous, intra-arterial and intracranial etc.

Workflow

References:

Le Guiner C, Moullier P, Arruda VR. Biodistribution and shedding of AAV vectors. Methods Mol Biol. 2011;807:339-59.
Rocha EM, Di Pasquale G, Riveros PP, Quinn K, Handelman B, Chiorini JA. Transduction, tropism, and biodistribution of AAV vectors in the lacrimal gland. Invest Ophthalmol Vis Sci. 2011 Dec 20;52(13):9567-72.
Schuster DJ, Dykstra JA, Riedl MS, Kitto KF, Belur LR, McIvor RS, Elde RP, Fairbanks CA, Vulchanova L. Biodistribution of adeno-associated virus serotype 9 (AAV9) vector after intrathecal and intravenous delivery in mouse. Front Neuroanat. 2014 Jun 10;8:42.
Liguore WA, Domire JS, Button D, Wang Y, Dufour BD, Srinivasan S, McBride JL. AAV-PHP.B Administration Results in a Differential Pattern of CNS Biodistribution in Non-human Primates Compared with Mice. Mol Ther. 2019 Nov 6;27(11):2018-2037.

* For research use only. Not intended for any clinical use.

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