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What are rAAV?
Adeno-Associated Virus (AAV): AAV is a small, non-enveloped virus that can infect both dividing and non-dividing cells. It is not known to cause disease in humans, making it a safe vector for gene therapy.
Recombinant AAV (rAAV): rAAV is genetically engineered AAV where the viral genes are replaced with therapeutic genes. This ensures that the virus cannot replicate, enhancing safety.
Advantages of rAAV
Safety: rAAV is non-pathogenic and elicits minimal immune responses compared to other viral vectors.
Long-term Expression: Once delivered, rAAV can provide stable and long-term expression of the therapeutic gene, especially in non-dividing cells.
Versatility: rAAV can infect a wide range of cell types and tissues, making it useful for various gene therapy applications.
Applications of rAAV
Gene Replacement Therapy: Used to treat genetic disorders by delivering functional copies of defective genes.
Gene Editing: Combined with CRISPR/Cas9 technology, rAAV can facilitate targeted gene editing.
Neurological Disorders: rAAV has been used to deliver therapeutic genes to the brain for diseases like Parkinson's and Alzheimer's.
Ophthalmic Diseases: rAAV vectors are used in treating inherited retinal diseases such as Leber's congenital amaurosis.
rAAV Production
Plasmid Transfection: rAAV is typically produced by transfecting cells with plasmids containing the therapeutic gene, AAV replication, and capsid genes, along with a helper plasmid providing necessary functions from adenovirus.
Purification: The rAAV particles are harvested from cell lysates and purified using techniques like iodixanol gradient centrifugation and affinity chromatography.
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