AAV (Adeno-associated virus)
Adeno-associated virus (AAV) is a small, non-enveloped virus that infects humans and some other primate species. It is not currently known to cause disease and consequently is of interest as a potential vector for gene therapy. The virus, which is naturally occurring, can infect both dividing and non-dividing cells and may incorporate its genome into that of the host cell. It is being researched for treatments of genetic disorders such as hemophilia, Duchenne muscular dystrophy, and others.
- Category
- Biological vector
- Molecular Formula
- Not applicable
- Mechanism of Action
- The AAV vector transduces cells by binding to specific receptors on the cell surface, entering the cell through endocytosis, and transporting its genome to the nucleus of the cell.
- Evidence Grade
- Clinical research
Other Names
Primary Benefits
- Potential for treating genetic disorders
- Infects both dividing and non-dividing cells
Recommended Dosage
| Type |
Not applicable |
| Description |
Dosage and administration are dependent on the specific gene therapy protocol and should be determined by a healthcare professional. |
Side Effects
- Immune responses
- Potential for off-target genetic effects
Precautions
- Not for individuals with active infections
- Pregnancy
- Immunocompromised individuals
Interactions
- Potential interactions with certain drugs that affect immune response
Key References
- https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3187629/
- https://www.nejm.org/doi/full/10.1056/NEJMoa1706198
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