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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

Product
Clonality
Application
Cat. No.
Quantity
ClonalityMonoclonal
ApplicationELISA, IHC
Cat. No.ABIN5954526
Quantity750 μL
ClonalityMonoclonal
ApplicationIF, IP, IHC, ELISA
Cat. No.ABIN933221
Quantity50 μg

AAV2 Antibodies

Product
Clonality
Application
Cat. No.
Quantity
ClonalityMonoclonal
ApplicationELISA, IHC
Cat. No.ABIN5954670
Quantity750 μL
ClonalityMonoclonal
ApplicationWB
Cat. No.ABIN1826239
Quantity50 μg

AAV3 Antibodies

Product
Clonality
Application
Cat. No.
Quantity
ClonalityMonoclonal
ApplicationWB
Cat. No.ABIN5954515
Quantity5 mL

AAV5 Antibodies

Product
Clonality
Application
Cat. No.
Quantity
ClonalityMonoclonal
ApplicationELISA, IHC
Cat. No.ABIN5954545
Quantity750 μL
ClonalityMonoclonal
ApplicationIF, IP, IHC, ELISA
Cat. No.ABIN933219
Quantity50 μg

AAV6 Antibodies

Product
Clonality
Application
Cat. No.
Quantity
ClonalityMonoclonal
ApplicationDB, EIA, IHC (fro), IF
Cat. No.ABIN1449389
Quantity5 mL

AAV8 Antibodies

Product
Clonality
Application
Cat. No.
Quantity
ClonalityMonoclonal
ApplicationIF, IP, Neut, IHC, ELISA
Cat. No.ABIN2132750
Quantity5 mL

AAV9 Antibodies

Product
Clonality
Application
Cat. No.
Quantity
ClonalityMonoclonal
ApplicationDB, IF, IP, IHC
Cat. No.ABIN2132751
Quantity5 mL
ClonalityMonoclonal
ApplicationDB, IF, IHC, ELISA
Cat. No.ABIN2132752
Quantity5 mL

References

  • (1) Naso MF, Tomkowicz B, Perry WL 3rd, Strohl WR. Adeno-Associated Virus (AAV) as a Vector for Gene Therapy. BioDrugs. 2017 Aug;31(4):317-334. doi: 10.1007/s40259-017-0234-5. PMID: 28669112; PMCID: PMC5548848.
  • (2) Wang D, Tai PWL, Gao G. Adeno-associated virus vector as a platform for gene therapy delivery. Nat Rev Drug Discov. 2019 May;18(5):358-378. doi: 10.1038/s41573-019-0012-9. PMID: 30710128; PMCID: PMC6927556.
  • (3) Xiao, W. et al. Gene therapy vectors based on adeno-associated virus type 1. J. Virol.73, 3994–4003 (1999).
  • (4) Manfredsson, F. P., Rising, A. C. & Mandel, R. J. AAV9: A potential blood-brain barrier buster. Molecular Therapy17, 403–405 (2009).
  • (5) Ai, J. et al. Adeno-associated virus serotype rh.10 displays strong muscle tropism following intraperitoneal delivery. Sci. Rep.7, (2017).
  • (6) Cheng, B. et al. Development of optimized AAV3 serotype vectors: Mechanism of high-efficiency transduction of human liver cancer cells. Gene Ther.19, 375–384 (2012).
  • (7) Mingozzi, F. & High, K. A. Immune responses to AAV vectors: Overcoming barriers to successful gene therapy. Blood122, 23–36 (2013).
  • (8) Doudna, J.A. The promise and challenge of therapeutic genome editing. Nature 578, 229–236 (2020)
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