[24] Adeno-Associated Viruses (AAVs) and Gene Therapy Flashcards
What are Adeno-Associated Viruses (AAVs)?
Small viruses that infect humans and some other primate species.
What is Gene Therapy?
A technique that modifies a person’s genes to treat or cure disease.
What is the function of AAVs in Gene Therapy?
AAVs can deliver genes to cells with minimal immune response, making them ideal for gene therapy applications.
What are the components of AAVs?
AAVs consist of a protein shell called a capsid that contains the genetic material.
What are the applications of AAVs and Gene Therapy?
They have numerous applications in treating genetic diseases such as hemophilia and Leber’s congenital amaurosis.
How does AAVs relate to Gene Therapy?
AAVs are used as vectors in Gene Therapy to deliver therapeutic genes to cells.
What are the benefits of using AAVs in Gene Therapy?
AAVs can deliver genes to cells with minimal immune response, which makes them ideal for gene therapy applications.
What diseases can be treated using AAVs and Gene Therapy?
Genetic diseases such as hemophilia and Leber’s congenital amaurosis can be treated using AAVs and Gene Therapy.
How do AAVs work in Gene Therapy?
AAVs work by delivering therapeutic genes to cells, thereby treating or curing diseases.
What role do AAVs play in research?
AAVs are widely used in research for gene delivery and gene therapy due to their ability to infect both dividing and non-dividing cells, and because they elicit a low immune response.
Why are AAVs ideal for research purposes?
AAVs are ideal for research because they can infect a wide range of species and tissues, elicit a low immune response, and have no known associated diseases.
How are AAVs used in genetic research?
AAVs are used as vectors in genetic research to deliver genes to cells, allowing researchers to study gene function, model diseases, and test gene therapies.
What makes AAVs effective for gene delivery in research?
AAVs are effective for gene delivery in research due to their ability to target specific tissue types and because they elicit a minimal immune response, allowing for long-term gene expression.
What is the significance of AAVs in the development of gene therapies?
AAVs have played a crucial role in the development of gene therapies due to their ability to deliver therapeutic genes to cells, their wide range of serotypes allowing for tissue-specific targeting, and their low pathogenicity.
Are there any limitations to using AAVs in research?
While AAVs are versatile tools, they have limitations such as a small packaging capacity for genetic material, possible immune responses in a subset of the population, and challenges in large-scale production.
