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Adeno-Associated Virus (AAV) Gene Delivery: Dissecting Molecular Interactions upon Cell Entry

期刊

VIRUSES-BASEL
卷 13, 期 7, 页码 -

出版社

MDPI
DOI: 10.3390/v13071336

关键词

adeno-associated virus; AAV; gene therapy; virus structure; AAV receptor; AAV attachment factor

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资金

  1. National Institute of Health (NIH) [R35 GM1222564]

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Human gene therapy has advanced significantly from the concept in the 20th century to a reality in the 21st century, with recombinant Adeno-Associated Virus (rAAV) being a major gene therapy vector. Research is continuously working on improving the safety and efficacy of rAAV through various AAV capsid modification strategies, focusing on factors such as neutralizing antibodies and receptor binding to enhance transduction efficiency. Advances in understanding molecular interactions during rAAV cell entry, in combination with improved capsid modulation strategies, will lead to the design of safer and more efficient rAAV gene therapy vectors.
Human gene therapy has advanced from twentieth-century conception to twenty-first-century reality. The recombinant Adeno-Associated Virus (rAAV) is a major gene therapy vector. Research continues to improve rAAV safety and efficacy using a variety of AAV capsid modification strategies. Significant factors influencing rAAV transduction efficiency include neutralizing antibodies, attachment factor interactions and receptor binding. Advances in understanding the molecular interactions during rAAV cell entry combined with improved capsid modulation strategies will help guide the design and engineering of safer and more efficient rAAV gene therapy vectors.

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