alexa AAV-Mediated Gene Therapy for Research and Therapeutic Purposes.
Genetics & Molecular Biology

Genetics & Molecular Biology

Journal of Molecular and Genetic Medicine

Author(s): Samulski RJ, Muzyczka N

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Abstract Adeno-associated virus (AAV) is a small, nonenveloped virus that was adapted 30 years ago for use as a gene transfer vehicle. It is capable of transducing a wide range of species and tissues in vivo with no evidence of toxicity, and it generates relatively mild innate and adaptive immune responses. We review the basic biology of AAV, the history of progress in AAV vector technology, and some of the clinical and research applications where AAV has shown success. This article was published in Annu Rev Virol and referenced in Journal of Molecular and Genetic Medicine

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