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• W3133555598 startingPage "683" @default.
• W3133555598 abstract "In the last two decades, recombinant adeno-associated virus has emerged as the most popular gene therapy vector. Recently AAV gene therapy has been approved by the FDA for the treatment of two rare genetic disorders, namely the early childhood blindness disease Leber congenital amaurosis and spinal muscular atrophy (SMA). As is the case for the treatment of SMA, if the AAV vector must be administered systemically, very high vector doses are often required for therapeutic efficacy. But higher vector doses inevitably increase the risk of adverse events. The tragic death of three children in a clinical trial to treat X-linked myotubular myopathy with an AAV vector has thrown this limitation into sharp relief. Regardless of the precise cause(s) that led to the death of the two children, it is critical that we develop better AAV vectors to achieve therapeutic levels of expression with lower vector doses. To transduce successfully a target cell, AAV has to overcome both systemic as well as cellular roadblocks. In this review, we discuss some of the most prominent cellular roadblocks that AAV must get past to deliver successfully its therapeutic payload. We also highlight recent advancements in our knowledge of AAV biology that can potentially be harnessed to improve AAV vector performance and thereby make AAV gene therapy safer." @default.
• W3133555598 created "2021-03-15" @default.
• W3133555598 creator A5070416578 @default.
• W3133555598 creator A5072736231 @default.
• W3133555598 date "2021-03-03" @default.
• W3133555598 modified "2023-10-17" @default.
• W3133555598 title "Intracellular trafficking of adeno-associated virus (AAV) vectors: challenges and future directions" @default.
• W3133555598 cites W1505196125 @default.
• W3133555598 cites W1630481045 @default.
• W3133555598 cites W1699517913 @default.
• W3133555598 cites W170859630 @default.
• W3133555598 cites W1756236371 @default.
• W3133555598 cites W1821381892 @default.
• W3133555598 cites W1871235281 @default.
• W3133555598 cites W191504613 @default.
• W3133555598 cites W1964861983 @default.
• W3133555598 cites W1966374792 @default.
• W3133555598 cites W1968006472 @default.
• W3133555598 cites W1968382713 @default.
• W3133555598 cites W1968469629 @default.
• W3133555598 cites W1974638173 @default.
• W3133555598 cites W1976210935 @default.
• W3133555598 cites W1977320723 @default.
• W3133555598 cites W1987453834 @default.
• W3133555598 cites W2000217017 @default.
• W3133555598 cites W2001674490 @default.
• W3133555598 cites W2001738143 @default.
• W3133555598 cites W2002565864 @default.
• W3133555598 cites W2003264964 @default.
• W3133555598 cites W2005754597 @default.
• W3133555598 cites W2007261697 @default.
• W3133555598 cites W2008289270 @default.
• W3133555598 cites W2009455163 @default.
• W3133555598 cites W2010666698 @default.
• W3133555598 cites W2014440721 @default.
• W3133555598 cites W2015426350 @default.
• W3133555598 cites W2015704490 @default.
• W3133555598 cites W2026642838 @default.
• W3133555598 cites W2029750163 @default.
• W3133555598 cites W2031617804 @default.
• W3133555598 cites W2040266789 @default.
• W3133555598 cites W204352406 @default.
• W3133555598 cites W2043835841 @default.
• W3133555598 cites W2046566574 @default.
• W3133555598 cites W2051442157 @default.
• W3133555598 cites W2052523589 @default.
• W3133555598 cites W2053820810 @default.
• W3133555598 cites W2057126633 @default.
• W3133555598 cites W2062840171 @default.
• W3133555598 cites W2063363340 @default.
• W3133555598 cites W2065639125 @default.
• W3133555598 cites W2066313345 @default.
• W3133555598 cites W2067189573 @default.
• W3133555598 cites W2070890881 @default.
• W3133555598 cites W2072901316 @default.
• W3133555598 cites W2075170026 @default.
• W3133555598 cites W2076046734 @default.
• W3133555598 cites W2076994245 @default.
• W3133555598 cites W2079090019 @default.
• W3133555598 cites W2079099220 @default.
• W3133555598 cites W2087100112 @default.
• W3133555598 cites W2089473921 @default.
• W3133555598 cites W2096579480 @default.
• W3133555598 cites W2097180939 @default.
• W3133555598 cites W2098414451 @default.
• W3133555598 cites W2098880579 @default.
• W3133555598 cites W2103904163 @default.
• W3133555598 cites W2104691616 @default.
• W3133555598 cites W2106326323 @default.
• W3133555598 cites W2108988659 @default.
• W3133555598 cites W2111051741 @default.
• W3133555598 cites W2111247770 @default.
• W3133555598 cites W2112808685 @default.
• W3133555598 cites W2113444875 @default.
• W3133555598 cites W2113637218 @default.
• W3133555598 cites W2120683337 @default.
• W3133555598 cites W2121034113 @default.
• W3133555598 cites W2122236774 @default.
• W3133555598 cites W2122304523 @default.
• W3133555598 cites W2125144482 @default.
• W3133555598 cites W2140596567 @default.
• W3133555598 cites W2141797403 @default.
• W3133555598 cites W2143529139 @default.
• W3133555598 cites W2147193337 @default.
• W3133555598 cites W2149491013 @default.
• W3133555598 cites W2151316131 @default.
• W3133555598 cites W2154329712 @default.
• W3133555598 cites W2155456837 @default.
• W3133555598 cites W2156683693 @default.
• W3133555598 cites W2157636936 @default.
• W3133555598 cites W2161591620 @default.
• W3133555598 cites W2164625869 @default.
• W3133555598 cites W2165254194 @default.
• W3133555598 cites W2165815741 @default.
• W3133555598 cites W2166618642 @default.
• W3133555598 cites W2168750366 @default.
• W3133555598 cites W2169091039 @default.
• W3133555598 cites W2169257419 @default.

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