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How the AAV Atlas can be used

Robert J. Gifford edited this page Jan 4, 2025 · 1 revision

What can the AAV Atlas be used for?

AAV-GLUE captures information about the genetic diversity of adeno-associated viruses (AAVs). It includes robust genotype assignments, homologous relationships, and detailed mutation information.

AAV-GLUE could have several significant uses, particularly in the context of gene therapy.

1. Gene Therapy Development and Optimization

Vector Design: Researchers can use the database to identify AAV strains with desirable properties for gene therapy, such as tissue tropism, immune evasion, and packaging capacity.

Capsid Engineering: By understanding the genetic variations and their impacts on AAV capsid properties, scientists can engineer capsids to improve transduction efficiency, reduce immune responses, and target specific tissues.

2. Clinical Applications

Patient-Specific Therapies: The database can help in designing AAV vectors tailored to individual patients, considering their specific immune profiles and the presence of pre-existing antibodies against certain AAV serotypes.

Predicting Treatment Outcomes: By correlating specific AAV genotypes and mutations with clinical outcomes, the database can assist in predicting the efficacy and safety of AAV-based therapies.

3. Basic Research

Evolutionary Studies: Researchers can study the evolutionary relationships between different AAV strains, understand their natural history, and identify evolutionary pressures that shape their genetic diversity.

Functional Genomics: The database can aid in investigating the functional impact of specific mutations on AAV biology, such as capsid stability, receptor binding, and intracellular trafficking.

4. Regulatory and Standardization

Quality Control: Standardized notation for mutations can help in quality control of AAV vectors, ensuring consistency and reliability in production and testing.

Regulatory Approval: Robust genotype assignments and mutation definitions can facilitate regulatory approval processes by providing detailed and standardized information on the vectors used in gene therapy.

5. Data Integration and Sharing

Public Health: The database can serve as a resource for public health monitoring, tracking the prevalence and spread of different AAV strains in the population.

Collaboration: It can provide a platform for collaboration among researchers, clinicians, and industry partners by offering a comprehensive and accessible repository of AAV genetic information.

6. Therapeutic Monitoring and Post-Treatment Analysis

Monitoring Viral Evolution: After gene therapy treatment, the database can be used to monitor any genetic changes in the AAV vectors, helping to understand potential evolution under therapeutic pressure.

Long-term Follow-up: Tracking mutations and genotypes in treated patients over time can provide insights into the long-term effects and stability of AAV-based therapies.

7. Educational and Training Resource

Training: The database can be used as a training tool for students and professionals in virology, gene therapy, and related fields.

Educational Outreach: It can help in disseminating knowledge about AAV biology and gene therapy to a broader audience, including healthcare professionals and the public.

8. Customized Applications

Diagnostic Tools: Development of diagnostic assays to detect specific AAV genotypes and mutations in biological samples, aiding in patient screening and monitoring.

Personalized Medicine: Tailoring gene therapy vectors to individual genetic profiles and specific disease contexts for personalized medical applications.