AAV stands for Adeno-Associated Virus. It is a small, non-enveloped virus that infects humans and other animals. AAV is considered a helper-dependent virus because it requires a helper virus, such as adenovirus or herpesvirus, to replicate and establish a productive infection. It is classified as a Parvoviridae family member and has a single-stranded DNA genome.
AAV is widely used in biomedical research and gene therapy applications due to its ability to efficiently infect both dividing and non-dividing cells. The virus has a broad host range and has been found to naturally infect various species without causing any known diseases or pathogenic effects.
In gene therapy, AAV vectors are frequently employed to deliver therapeutic genes into target cells. These vectors are genetically modified versions of the virus that have had their own viral genes replaced with therapeutic genes of interest. AAV vectors offer many advantages, including long-lasting expression of the delivered genes and a relatively low immunogenicity, making them ideal for potential gene therapy treatments.
As a viral vector, AAV offers versatility and has been extensively studied for its safety profile. Extensive research has been conducted to optimize its packaging capacity, target cell specificity, and immune response evasion. AAV-based gene therapies have shown promise in treating a variety of genetic disorders, including inherited retinal diseases, muscular dystrophies, and neurodegenerative disorders.
Overall, AAV is a versatile and widely used viral vector that has opened up new avenues in gene therapy and biomedical research. Its unique characteristics make it an attractive candidate for developing safe and effective treatments for various genetic diseases.
