Adeno-Associated Virus

An Adeno-Associated Virus is a small DNA dependoparvovirus belonging to the Parvoviridae family (characterized by its dependence on helper viruses for replication and its non-pathogenic nature in humans).

• Context:
  • It can typically contain a Single-Stranded DNA Genome of approximately 4.7 kilobases in length.
  • It can typically require Helper Virus functions from adenoviruses or herpesviruses to complete its viral replication cycle.
  • It can typically package its viral genome within an icosahedral capsid composed of viral proteins VP1, VP2, and VP3.
  • It can typically enter host cells through binding to specific cell surface receptors including heparan sulfate proteoglycans.
  • It can typically persist in host tissues through episomal maintenance or site-specific integration into the human genome.
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  • It can often exhibit Tissue Tropism based on its viral serotype and capsid protein composition.
  • It can often establish Latent Infection in human tissues without causing clinical disease.
  • It can often integrate at a specific location called AAVS1 on human chromosome 19 when wild-type AAV integration occurs.
  • It can often trigger minimal Immune Response compared to other viral vectors used in gene therapy.
  • It can often cross the Blood-Brain Barrier in certain serotypes like AAV9 to reach central nervous system tissues.
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  • It can range from being a Wild-Type Adeno-associated Virus to being a Recombinant Adeno-associated Virus, depending on its genome composition.
  • It can range from being a Simple Adeno-associated Virus to being an Engineered Adeno-associated Virus, depending on its capsid modification.
  • It can range from being a Natural Adeno-associated Virus Serotype to being a Synthetic Adeno-associated Virus Variant, depending on its origin.
  • It can range from being a Low-Titer Adeno-associated Virus to being a High-Titer Adeno-associated Virus, depending on its production method.
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  • It can have Genetic Structure including inverted terminal repeats that flank the viral genes and serve as origin of replication.
  • It can provide Therapeutic Potential through its use as a gene therapy vector for genetic disease treatment.
  • It can demonstrate Clinical Safety through its non-pathogenic nature and low immunogenicity profile.
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• Examples:
  • Adeno-associated Virus Serotypes, such as:
    • Natural Adeno-associated Virus Serotypes, such as:
      • Adeno-associated Virus Serotype 2 with liver and central nervous system tropism.
      • Adeno-associated Virus Serotype 8 with strong liver tropism.
      • Adeno-associated Virus Serotype 9 with cardiac and systemic tissue tropism.
    • Engineered Adeno-associated Virus Serotypes, such as:
      • Adeno-associated Virus Anc80 for inner ear targeting.
      • Adeno-associated Virus ShH19 for enhanced musculoskeletal tropism.
  • Adeno-associated Virus Vectors, such as:
    • Self-Complementary Adeno-associated Virus Vectors for enhanced gene expression.
    • Single-Stranded Adeno-associated Virus Vectors for larger transgene capacity.
    • Dual Adeno-associated Virus Vectors for delivery of oversized genes.
  • Adeno-associated Virus Manufacturing Platforms, such as:
    • HEK293 Cell-Based Adeno-associated Virus Production for research-scale manufacturing.
    • Baculovirus-Insect Cell Adeno-associated Virus Production for large-scale manufacturing.
    • Suspension Cell Adeno-associated Virus Production for industrial-scale manufacturing.
  • Adeno-associated Virus Clinical Applications, such as:
    • Ocular Adeno-associated Virus Therapy for inherited retinal disease treatment.
    • Hepatic Adeno-associated Virus Therapy for metabolic disorder treatment.
    • Neurologic Adeno-associated Virus Therapy for neurodegenerative disease treatment.
    • Muscular Adeno-associated Virus Therapy for muscular dystrophy treatment.
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• Counter-Examples:
  • Adenovirus, which is pathogenic and serves as a helper virus for adeno-associated virus replication rather than requiring assistance itself.
  • Lentivirus, which is an RNA virus capable of independent replication and integration throughout the genome rather than at specific sites.
  • Autonomous Parvovirus, which can replicate independently without helper virus functions unlike adeno-associated virus.
  • Herpes Simplex Virus, which has a large DNA genome and complex structure compared to the simple genome and icosahedral structure of adeno-associated virus.
• See: Dependoparvovirus, Viral Vector, Gene Therapy Vector, Helper Virus, Viral Capsid.

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