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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 AAV

Below you will find an overview of some important target organs for AAV(2). We offer a variety of hand-picked antibodies and proteins for your research. By clicking on the links you will be directed to the available products.

Liver(2),(5),(6),(7)

  • MPS: +
  • OTC deficiency:
  • Crigler-Najjar syndrome:
  • Haemophilia A + B: + +

Muscle(2),(4),(7)

  • A1At deficiency:
  • CMT1A:
  • HIV infections: +
  • Dysferlinopathy: AAVrh.74
  • Pompe disease: +
  • X-linked MTM:
  • DMD: AAVrh.74 +

Brain(2),(3),(4)

  • AADC deficiency:
  • Batten disease (CLN6):
  • MPS-IIIB:
  • Parkinson disease:

Eye(2)

  • Achromatopsia: +
  • Choroideraemia:
  • LCA: +
  • LHON:
  • RP (RLBP1):
  • X-linked RP:
  • X-linked retinoschisis: +

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

Produit
Clonalité
Application
N° du produit
Quantité
Fiche technique
Clonalité Monoclonal
Application IF, IP, IHC, ELISA
N° du produit ABIN933221
Quantité 50 μg
Fiche technique Fiche technique
Clonalité Monoclonal
Application ELISA, IHC
N° du produit ABIN5954526
Quantité 750 μL
Fiche technique Fiche technique

AAV2 Antibodies

Produit
Clonalité
Application
N° du produit
Quantité
Fiche technique
Clonalité Monoclonal
Application ELISA, IHC
N° du produit ABIN5954670
Quantité 750 μL
Fiche technique Fiche technique
Clonalité Monoclonal
Application WB
N° du produit ABIN1826239
Quantité 50 μg
Fiche technique Fiche technique

AAV3 Antibodies

Produit
Clonalité
Application
N° du produit
Quantité
Fiche technique
Clonalité Monoclonal
Application WB
N° du produit ABIN5954515
Quantité 5 mL
Fiche technique Fiche technique

AAV5 Antibodies

Produit
Clonalité
Application
N° du produit
Quantité
Fiche technique
Clonalité Monoclonal
Application IF, IP, IHC, ELISA
N° du produit ABIN933219
Quantité 50 μg
Fiche technique Fiche technique
Clonalité Monoclonal
Application ELISA, IHC
N° du produit ABIN5954545
Quantité 750 μL
Fiche technique Fiche technique

AAV6 Antibodies

Produit
Clonalité
Application
N° du produit
Quantité
Fiche technique
Clonalité Monoclonal
Application DB, EIA, IHC (fro), IF
N° du produit ABIN1449389
Quantité 5 mL
Fiche technique Fiche technique

AAV8 Antibodies

Produit
Clonalité
Application
N° du produit
Quantité
Fiche technique
Clonalité Monoclonal
Application IF, IP, Neut, IHC, ELISA
N° du produit ABIN2132750
Quantité 5 mL
Fiche technique Fiche technique

AAV9 Antibodies

Produit
Clonalité
Application
N° du produit
Quantité
Fiche technique
Clonalité Monoclonal
Application DB, IF, IHC, ELISA
N° du produit ABIN2132752
Quantité 5 mL
Fiche technique Fiche technique
Clonalité Monoclonal
Application DB, IF, IP, IHC
N° du produit ABIN2132751
Quantité 5 mL
Fiche technique Fiche technique

References

  1. Naso, Tomkowicz, Perry, Strohl: "Adeno-Associated Virus (AAV) as a Vector for Gene Therapy." dans: 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." dans: 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." dans: Journal of virology, Vol. 73, Issue 5, pp. 3994-4003, (1999) (PubMed).
  4. Manfredsson, Rising, Mandel: "AAV9: a potential blood-brain barrier buster." dans: 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." dans: 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." dans: 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." dans: Blood, Vol. 122, Issue 1, pp. 23-36, (2013) (PubMed).
  8. Doudna: "The promise and challenge of therapeutic genome editing." dans: 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." dans: Nature reviews. Drug discovery, Vol. 20, Issue 3, pp. 173-174, (2021) (PubMed).
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