Adeno-Associated Virus (AAV) vectors in gene therapy

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Human gene therapy offers significant potential for curing various genetic diseases, requiring two key elements: the cloning and characterization of the affected gene, and a suitable delivery vector for a healthy gene copy. An effective vector should possess several desirable characteristics, including the ability to carry the intact gene (either genomic or cDNA) stably, introduce the gene into the target cell type, express the gene in a regulated manner over an extended period, and minimize toxicity to the recipient. Other considerations include the frequency of cell transformation and the capacity to introduce genes into nondividing stem cells. Various animal viruses have been explored as potential vectors, but none meet all the necessary criteria. For instance, retroviruses face challenges in propagation at sufficient titers, do not integrate into nondividing cells, and raise oncogenic concerns due to their ability to integrate at multiple genomic sites, posing risks of insertional mutagenesis. Additionally, genes delivered by retroviral vectors often have limited expression duration. Adenoviruses have also been utilized as vectors in experimental models.