Course Overview: Adeno-Associated Virus (AAV) Vectors
Course Title:
Adeno-Associated Virus (AAV) Biology, Engineering, and Applications in Gene Therapy
Course Description:
This course provides a comprehensive introduction to Adeno-Associated Virus (AAV), one of the most widely used viral vectors in modern gene therapy. Students will explore AAV biology, vector design, production methods, safety considerations, regulatory requirements, and therapeutic applications. The course blends molecular virology, biotechnology, and translational medicine, preparing learners to understand and evaluate AAV-based therapeutic strategies.
Learning Objectives:
By the end of this course, students will be able to:
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Explain the basic biology and life cycle of AAV.
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Describe AAV vector design, including promoters, capsid variants, and genome packaging.
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Compare AAV serotypes, tropisms, and engineered capsids.
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Understand production, purification, and quantification techniques.
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Evaluate preclinical and clinical applications of AAV vectors.
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Assess immune responses, biosafety, and regulatory considerations.
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Analyze case studies of FDA-approved AAV gene therapies.
Course Modules
Module 1: Introduction to AAV Biology
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Discovery and natural biology
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AAV structure and genome organization
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Rep and Cap proteins
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Dependence on helper viruses
Module 2: AAV Vector Engineering
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Recombinant AAV (rAAV) design
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Packaging capacity limitations
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Use of promoters, enhancers, and regulatory elements
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Self-complementary AAV (scAAV)
Module 3: AAV Serotypes & Capsid Engineering
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Natural serotypes and tissue tropism
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Rational and directed evolution capsid engineering
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Immune evasion strategies
Module 4: AAV Production & Purification
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HEK293-based transient transfection systems
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Helper virus–based systems
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Purification techniques (CsCl, iodixanol, chromatography)
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Quality control and assay methods
Module 5: Preclinical Studies
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Animal models
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Biodistribution and transduction efficiency
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Dose considerations
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Toxicology
Module 6: Clinical Applications of AAV
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Approved therapies (e.g., Luxturna, Zolgensma)
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Ongoing clinical trials
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Organ-specific delivery: CNS, liver, retina, muscle
Module 7: Safety & Regulatory Considerations
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Immune responses to AAV
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Off-target effects
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Manufacturing guidelines (GMP)
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Regulatory pathways (FDA/EMA)
Module 8: Future Directions
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Next-generation capsids
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Non-viral gene transfer
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Genome editing with AAV (CRISPR delivery)
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Ethical and cost considerations
Target Audience
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Biotechnology and biomedical engineering students
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Researchers entering gene therapy
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Professionals in pharma/biotech
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Clinicians interested in translational medicine
- Teacher: Immaculate Cheruto
- Teacher: Richard Yahuma