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. 2013 Nov;21(11):1976-81.
doi: 10.1038/mt.2013.226.

Birth of a new therapeutic platform: 47 years of adeno-associated virus biology from virus discovery to licensed gene therapy

Terence R Flotte  1
Affiliations

Birth of a new therapeutic platform: 47 years of adeno-associated virus biology from virus discovery to licensed gene therapy

Terence R Flotte. Mol Ther. 2013 Nov.
No abstract available

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Figures

Figure 1
Figure 1
Time line of major milestones in AAV biology and vectorology. The 47 years between the discovery of adeno-associated virus (AAV) and the licensure of Glybera are depicted in three intervals, each approximately 15 years long (1965–1982, 1982–1997, and 1997–2012), with each individually marked segment representing approximately 5 years. IM, intramuscular; ITR, inverted terminal repeat.
Figure 2
Figure 2
Figure from a 1969 publication on adeno-associated virus (AAV) biology. The illustration depicts an AAV's unusual property of being packaged as single-stranded DNA with equal proportions of plus and minus sense strands. Such studies spurred interest in AAV long before the advent of human gene therapy. Reprinted with permission from ref. .
Figure 3
Figure 3
Evidence of efficacy in a human gene therapy trial. The trial used a rAAV2-RPE65 vector to treat patients with the autosomal recessive blinding disorder Leber's congenital amaurosis (LCA). The several-log10-fold change in light sensitivity in the injected portions of the treated retinas (indicated by stars) of three patients (P1, P2, P3) indicates benefit to visual function in these patients. On the opposite side of the panel are the light-sensitivity results from the contralateral eyes (P1, P2, P3) and from an untreated patient (RPE65-LCA). Reprinted with permission from ref. .
Figure 4
Figure 4
The biodiversity of primate-derived adeno-associated virus (AAV) genomic variants. The variants (shown within clades here), which were identified in in the landmark 2004 article by Gao et al.,98 may provide many new potential capsid vehicles for therapeutic gene transfer in the future. Reprinted with permission from ref. .
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