Gene therapy for retinitis pigmentosa and Leber congenital amaurosis caused by defects in AIPL1 : effective rescue of mouse models of partial and complete Aipl1 deficiency using AAV2/2 and AAV2/8 vectors

By Tan Mei Hong   Smith Alexander J.   Pawlyk Basil   Xu Xiaoyun   Liu Xiaoqing   Bainbridge James B.   Basche Mark   McIntosh Jenny   Tran Hoai Viet   Nathwani Amit   Li Tiansen   Ali Robin R.  

Human Molecular Genetics, Vol. 18, Iss. 12, 2009-06 ,pp. : 2099-2114

Oxford University Press

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The Leber congenital amaurosis gene product AIPL1 is localized exclusively in rod photoreceptors of the adult human retina

By Spuy J.v.d.   Chapple J.P.   Clark B.J.   Luthert P.J.   Sethi C.S.   Cheetham M.E.  

Human Molecular Genetics, Vol. 11, Iss. 7, 2002-04 ,pp. : 823-831

Oxford University Press

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Gene therapy using self-complementary Y733F capsid mutant AAV2/8 restores vision in a model of early onset Leber congenital amaurosis

By Ku Cristy A.   Chiodo Vince A.   Boye Sanford L.   Goldberg Andrew F.X.   Li Tiansen   Hauswirth William W.   Ramamurthy Visvanathan  

Human Molecular Genetics, Vol. 20, Iss. 23, 2011-12 ,pp. : 4569-4581

Oxford University Press

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The Leber congenital amaurosis protein, AIPL1, is needed for the viability and functioning of cone photoreceptor cells

By Kirschman Lindsay T.   Kolandaivelu Saravanan   Frederick Jeanne M.   Dang Loan   Goldberg Andrew F.X.   Baehr Wolfgang   Ramamurthy Visvanathan  

Human Molecular Genetics, Vol. 19, Iss. 6, 2010-03 ,pp. : 1076-1087

Oxford University Press

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Gene therapy restores vision and delays degeneration in the CNGB1 −/− mouse model of retinitis pigmentosa

By Koch Susanne   Sothilingam Vithiyanjali   Garcia Garrido Marina   Tanimoto Naoyuki   Becirovic Elvir   Koch Fred   Seide Christina   Beck Susanne C.   Seeliger Mathias W.   Biel Martin   Mühlfriedel Regine   Michalakis Stylianos  

Human Molecular Genetics, Vol. 21, Iss. 20, 2012-10 ,pp. : 4486-4496

Oxford University Press

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