AAV Vectors

Definition

AAV vectors, or adeno-associated virus vectors, are a type of viral vector used in gene therapy to deliver genetic material into cells. They are derived from adeno-associated viruses, which are non-pathogenic and have a natural ability to infect a wide range of cell types without causing disease. Their unique properties make AAV vectors attractive for gene delivery, as they can achieve stable integration into the host genome and exhibit low immunogenicity.

5 Must Know Facts For Your Next Test

1. AAV vectors can carry a relatively small amount of genetic material, typically up to 4.7 kb, which limits the size of the genes that can be delivered.
2. These vectors can transduce both dividing and non-dividing cells, making them versatile for various applications in gene therapy.
3. AAV vectors have a low risk of causing an immune response, allowing for repeated administration in patients if necessary.
4. They can integrate into specific sites within the genome or remain as episomes, enabling long-term expression of the delivered gene.
5. AAV vectors have been successfully used in clinical trials for conditions such as hemophilia and retinal diseases, showcasing their potential in regenerative medicine.

Review Questions

• How do AAV vectors differ from other viral vectors in terms of their application in gene therapy?
  • AAV vectors differ from other viral vectors like lentivirus and retrovirus primarily in their safety profile and ability to integrate into the host genome. While lentivirus can integrate into any part of the genome and retroviruses typically require dividing cells for integration, AAV vectors are less likely to trigger immune responses and can transduce both dividing and non-dividing cells. This makes AAV vectors particularly advantageous for targeting tissues such as muscle or neurons where non-dividing cells are prevalent.
• Discuss the advantages and limitations of using AAV vectors for gene delivery compared to non-viral methods.
  • Using AAV vectors for gene delivery offers several advantages over non-viral methods, including efficient transduction of target cells and long-term expression of the therapeutic gene. They also exhibit low immunogenicity, which is beneficial for repeated treatments. However, their limitations include the relatively small cargo capacity for genetic material and potential challenges with large-scale production. Non-viral methods, such as liposomes or electroporation, may allow for larger DNA constructs but generally result in lower transfection efficiency.
• Evaluate the current clinical applications of AAV vectors in gene therapy and predict future trends based on existing research.
  • Currently, AAV vectors are being used in clinical applications such as treating genetic disorders like hemophilia and inherited retinal diseases. Ongoing research continues to expand their use into areas such as muscular dystrophy and neurodegenerative diseases. Future trends may include engineering AAV vectors with enhanced tropism to target specific cell types more effectively and developing next-generation vectors that can accommodate larger genes or multiple genes simultaneously. This innovation could significantly broaden the scope of conditions that gene therapy could address.

Related terms

Gene Therapy: A technique that modifies a person's genes to treat or prevent disease, often using vectors to deliver therapeutic genes.

Viral Vectors: Tools derived from viruses that are used to deliver genetic material into cells, including AAV, lentivirus, and retrovirus vectors.

Transduction: The process by which foreign DNA is introduced into a cell using a vector, allowing for gene expression or functional modifications.