AAV in Gene Therapy

Adeno-Associated Virus is a protein shell surrounding and protecting a small, single-stranded DNA genome of approximately 4.8 kilobases. AAV belongs to the parvovirus family and is dependent on co-infection with other viruses, mainly adenoviruses, in order to replicate.⁽¹⁾ AAV vectors are the leading platform for gene delivery for the treatment of a variety of human diseases. Recent advances in developing clinically desirable AAV capsids, optimizing genome designs and harnessing revolutionary biotechnologies have contributed substantially to the growth of the gene therapy field.⁽²⁾

AAVs are the most commonly used method for delivering gene-editing tools like CRISPR-Cas9. The use of AAVs in gene-replacement therapy approved by the FDA for patients suffering from spinal muscular atrophy and congential blindness. The advantages of AAV are reduced risk of genomic integration, broad tissue targeting possibilities, clinically manageable immunogenicity. Current challenges are low genetic "cargo load" and reduced efficiency when using second AAV vector to increase "cargo load", as well as undesired off-target effects due to long-term expression of gene-editing molecules and scalability issues regarding AAV manufacturing.⁽⁸⁾

Important Target Organs for AAVs

Below you will find an overview of some important target organs for AAVs⁽²⁾. AAV2 is the most frequently used serotype. The most safety and efficacy data - from more than 40 completed trials - is available for this serotype as well. for delivery to the central nervous system (CNS) AAV8 and AAV9 are being used more and more as gene therapy gains traction for CNS diseases.⁽⁹⁾

Monoclonal Antibodies against AAV Targets

We offer a unique selection of Adeno-Associated Virus (AAV) antibodies for gene therapy research. Click on the links to see more product details.

AAV1 Antibodies

AAV2 Antibodies

AAV3 Antibodies

AAV5 Antibodies

AAV6 Antibodies

AAV8 Antibodies

AAV9 Antibodies

References

1. Naso, Tomkowicz, Perry, Strohl: "Adeno-Associated Virus (AAV) as a Vector for Gene Therapy." in: BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy, Vol. 31, Issue 4, pp. 317-334, (2018) (PubMed).
2. Wang, Tai, Gao: "Adeno-associated virus vector as a platform for gene therapy delivery." in: Nature reviews. Drug discovery, Vol. 18, Issue 5, pp. 358-378, (2019) (PubMed).
3. Xiao, Chirmule, Berta, McCullough, Gao, Wilson: "Gene therapy vectors based on adeno-associated virus type 1." in: Journal of virology, Vol. 73, Issue 5, pp. 3994-4003, (1999) (PubMed).
4. Manfredsson, Rising, Mandel: "AAV9: a potential blood-brain barrier buster." in: Molecular therapy : the journal of the American Society of Gene Therapy, Vol. 17, Issue 3, pp. 403-5, (2009) (PubMed).
5. Ai, Li, Gessler, Su, Wei, Li, Gao: "Adeno-associated virus serotype rh.10 displays strong muscle tropism following intraperitoneal delivery." in: Scientific reports, Vol. 7, pp. 40336, (2018) (PubMed).
6. Cheng, Ling, Dai, Lu, Glushakova, Gee, McGoogan, Aslanidi, Park, Stacpoole, Siemann, Liu, Srivastava, Ling: "Development of optimized AAV3 serotype vectors: mechanism of high-efficiency transduction of human liver cancer cells." in: Gene therapy, Vol. 19, Issue 4, pp. 375-84, (2012) (PubMed).
7. Mingozzi, High: "Immune responses to AAV vectors: overcoming barriers to successful gene therapy." in: Blood, Vol. 122, Issue 1, pp. 23-36, (2013) (PubMed).
8. Doudna: "The promise and challenge of therapeutic genome editing." in: Nature, Vol. 578, Issue 7794, pp. 229-236, (2020) (PubMed).
9. Kuzmin, Shutova, Johnston, Smith, Fedorin, Kukushkin, van der Loo, Johnstone: "The clinical landscape for AAV gene therapies." in: Nature reviews. Drug discovery, Vol. 20, Issue 3, pp. 173-174, (2021) (PubMed).

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