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- W2897417813 abstract "Voretigene neparvovec‐rzyl was recently approved for the treatment of Leber congenital amaurosis, and the use of gene therapy for eye disease is attracting even greater interest. The eye has immune privileged status, is easily accessible, requires a reduced dosage of therapy due to its size and is highly compartmentalized, significantly reducing systemic spread. Adeno‐associated virus (AAV), with its low pathogenicity, prolonged expression profile and ability to transduce multiple cell types, has become the leading gene therapy vector. Target diseases have moved beyond currently untreatable inherited dystrophies to common, partially treatable acquired conditions such as exudative age‐related macular degeneration and glaucoma, but use of the technology in these conditions imposes added obligations for caution in vector design. This review discusses the current status of AAV gene therapy trials in genetic and acquired ocular diseases, and explores new scientific developments, which could help ensure effective and safe use of the therapy in the future." @default.
- W2897417813 created "2018-10-26" @default.
- W2897417813 creator A5005203967 @default.
- W2897417813 creator A5024604818 @default.
- W2897417813 creator A5027492632 @default.
- W2897417813 creator A5048307488 @default.
- W2897417813 creator A5051792109 @default.
- W2897417813 creator A5076911708 @default.
- W2897417813 date "2019-01-02" @default.
- W2897417813 modified "2023-10-16" @default.
- W2897417813 title "Gene therapy and the adeno‐associated virus in the treatment of genetic and acquired ophthalmic diseases in humans: Trials, future directions and safety considerations" @default.
- W2897417813 cites W1429161016 @default.
- W2897417813 cites W1526659024 @default.
- W2897417813 cites W1569820390 @default.
- W2897417813 cites W1599588446 @default.
- W2897417813 cites W1697173196 @default.
- W2897417813 cites W1755815992 @default.
- W2897417813 cites W1799877731 @default.
- W2897417813 cites W1915008606 @default.
- W2897417813 cites W1929485725 @default.
- W2897417813 cites W1966986920 @default.
- W2897417813 cites W1969811289 @default.
- W2897417813 cites W1970791968 @default.
- W2897417813 cites W1971470549 @default.
- W2897417813 cites W1982197481 @default.
- W2897417813 cites W1982690958 @default.
- W2897417813 cites W1984425391 @default.
- W2897417813 cites W1992015063 @default.
- W2897417813 cites W1996875707 @default.
- W2897417813 cites W1997031423 @default.
- W2897417813 cites W1998130034 @default.
- W2897417813 cites W1999610747 @default.
- W2897417813 cites W2000229924 @default.
- W2897417813 cites W2001032066 @default.
- W2897417813 cites W2010048790 @default.
- W2897417813 cites W2010207266 @default.
- W2897417813 cites W2012134951 @default.
- W2897417813 cites W2012902380 @default.
- W2897417813 cites W2017638920 @default.
- W2897417813 cites W2020815323 @default.
- W2897417813 cites W2022084143 @default.
- W2897417813 cites W2023962213 @default.
- W2897417813 cites W2031303778 @default.
- W2897417813 cites W2034883885 @default.
- W2897417813 cites W2035101755 @default.
- W2897417813 cites W2035534672 @default.
- W2897417813 cites W2036166005 @default.
- W2897417813 cites W2036846364 @default.
- W2897417813 cites W2037348124 @default.
- W2897417813 cites W2038567516 @default.
- W2897417813 cites W2041189307 @default.
- W2897417813 cites W2041395551 @default.
- W2897417813 cites W2042523510 @default.
- W2897417813 cites W2046047442 @default.
- W2897417813 cites W2047747556 @default.
- W2897417813 cites W2048313278 @default.
- W2897417813 cites W2049484831 @default.
- W2897417813 cites W2054158396 @default.
- W2897417813 cites W2054303973 @default.
- W2897417813 cites W2056602419 @default.
- W2897417813 cites W2057314625 @default.
- W2897417813 cites W2061331516 @default.
- W2897417813 cites W2062308675 @default.
- W2897417813 cites W2066080711 @default.
- W2897417813 cites W2070726099 @default.
- W2897417813 cites W2071404720 @default.
- W2897417813 cites W2071623922 @default.
- W2897417813 cites W2074042512 @default.
- W2897417813 cites W2074239537 @default.
- W2897417813 cites W2075794747 @default.
- W2897417813 cites W2079032125 @default.
- W2897417813 cites W2082025265 @default.
- W2897417813 cites W2084637683 @default.
- W2897417813 cites W2086498377 @default.
- W2897417813 cites W2091415724 @default.
- W2897417813 cites W2092670526 @default.
- W2897417813 cites W2093150080 @default.
- W2897417813 cites W2094688362 @default.
- W2897417813 cites W2100821349 @default.
- W2897417813 cites W2101049277 @default.
- W2897417813 cites W2109335923 @default.
- W2897417813 cites W2111637908 @default.
- W2897417813 cites W2114564223 @default.
- W2897417813 cites W2114741749 @default.
- W2897417813 cites W2116916434 @default.
- W2897417813 cites W2120723151 @default.
- W2897417813 cites W2123950745 @default.
- W2897417813 cites W2126115955 @default.
- W2897417813 cites W2132060134 @default.
- W2897417813 cites W2136083637 @default.
- W2897417813 cites W2137222303 @default.
- W2897417813 cites W2137826201 @default.
- W2897417813 cites W2139489264 @default.
- W2897417813 cites W2140135936 @default.
- W2897417813 cites W2141354154 @default.
- W2897417813 cites W2142695353 @default.
- W2897417813 cites W2145775145 @default.
- W2897417813 cites W2146356499 @default.