Adeno-associated virus (AAV) vectors are crucial in gene therapy, delivering genes to treat genetic diseases. Luxturna and Zolgensma exemplify their use in restoring vision and treating spinal muscular atrophy, respectively. AAV vectors face challenges like limited genetic payload and immune responses, but advancements in vector design and production aim to overcome these hurdles.
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AAV vectors are advantageous for repeated administration in clinical settings due to their minimal immunogenicity and ability to target a diverse array of cell types
AAV vectors are limited by their genetic payload capacity and there is a risk of eliciting immune responses that can diminish their therapeutic efficacy
Ongoing research is focused on addressing challenges such as pre-existing immunity and limited capacity, with innovations in vector design and production techniques being developed to enhance the yield, purity, and potency of AAV vectors
Luxturna and Zolgensma are FDA-approved gene therapies that use AAV vectors to correct gene defects in inherited retinal disease and spinal muscular atrophy, respectively
AAV vectors are being explored for their potential use in creating vaccines for diseases such as COVID-19
The selection of an AAV vector for gene therapy is guided by the target tissue and the desired persistence of gene expression, with multiple serotypes available for targeting specific organs or cell types
AAV vectors should be handled in a Class II Biosafety Cabinet and proper use of Personal Protective Equipment (PPE) is mandatory to protect against aerosol exposure
Proper disposal procedures for AAV-containing materials and decontamination of work surfaces are essential to prevent environmental contamination
Training for laboratory personnel is imperative to ensure understanding of biosafety practices and to maintain a secure working environment, with prompt response to any incidents of exposure or contamination necessary to safeguard health and safety
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Origin of AAV vectors
Derived from a benign virus, modified for gene therapy.
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AAV vector immunogenicity
Engineered to be minimally immunogenic, suitable for repeated use.
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AAV vector tissue tropism
Broad tissue tropism allows targeting of various cell types.
