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Author Spotlight: Optimizing Digital Droplet PCR Method for Accurate Adeno-Associated Viral Genome Quantification
Published on: October 11, 2024
AAV-Mediated Gene Therapy for Research and Therapeutic Purposes
R Jude Samulski1, Nicholas Muzyczka2
1Gene Therapy Center, University of North Carolina, Chapel Hill, North Carolina 27599;
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.
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