Adeno-Associated Virus (AAV)

Definition

Adeno-Associated Virus (AAV) is a small virus that is not associated with any known disease in humans and has gained significant attention for its potential as a gene delivery vector. AAV is used to deliver therapeutic genes into cells, offering a promising approach for treating genetic disorders, as it can integrate into the host genome and provide long-term expression of the delivered gene. Its unique ability to be non-pathogenic and its favorable safety profile make AAV an attractive option for various gene therapy applications.

5 Must Know Facts For Your Next Test

1. AAV is a member of the Parvoviridae family and has a single-stranded DNA genome, which makes it distinct from many other viral vectors used in gene delivery.
2. One of the main advantages of using AAV is its ability to achieve stable and long-term gene expression without causing harmful immune responses in the host.
3. AAV can infect both dividing and non-dividing cells, making it versatile for various types of tissues and conditions.
4. Different serotypes of AAV exist, each with varying tropisms for different tissues, allowing for targeted gene delivery based on the specific needs of treatment.
5. Clinical trials have demonstrated the efficacy of AAV-based therapies for conditions like hemophilia and certain types of inherited retinal diseases.

Review Questions

• How does the structure of AAV contribute to its effectiveness as a gene delivery vector?
  • The small size and simple structure of AAV allow it to efficiently package genetic material and deliver it into target cells. Its single-stranded DNA genome is less likely to provoke an immune response, which helps ensure stable transduction. Additionally, AAV's ability to integrate into the host genome allows for prolonged expression of therapeutic genes, enhancing its effectiveness compared to other vectors that might not provide long-lasting results.
• Discuss the advantages and limitations of using AAV in gene therapy compared to other viral vectors.
  • AAV offers several advantages over other viral vectors, including its non-pathogenic nature and lower risk of causing an immune response, which promotes long-term expression of the delivered gene. It can infect a wide range of cell types, including non-dividing cells, making it suitable for diverse applications. However, limitations include its relatively small packaging capacity for genetic material and potential pre-existing immunity in some patients due to natural exposure to AAV, which could hinder its effectiveness in certain cases.
• Evaluate the role of AAV in the future of personalized medicine and its potential impact on treating genetic disorders.
  • AAV holds a pivotal role in the future of personalized medicine due to its ability to deliver specific therapeutic genes tailored to individual genetic profiles. This flexibility allows researchers to develop targeted therapies for various genetic disorders with greater precision. As advancements continue in vector engineering and understanding host interactions, AAV could significantly improve treatment outcomes and pave the way for curative approaches in conditions previously deemed untreatable.

Related terms

Gene Therapy: A technique that modifies or replaces genes to treat or prevent disease, often using vectors like AAV to deliver therapeutic genes.

Vector: A vehicle used to transfer genetic material into a target cell; AAV serves as one of the most widely used vectors in gene therapy.

Transduction: The process by which a vector, such as AAV, introduces foreign DNA into a host cell, leading to gene expression.