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. 19, Iss. 4, 2010-02 ,pp. : 735-735

Oxford University Press

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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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Gene therapy provides long-term visual function in a pre-clinical model of retinitis pigmentosa

By Wert Katherine J.   Davis Richard J.   Sancho-Pelluz Javier   Nishina Patsy M.   Tsang Stephen H.  

Human Molecular Genetics, Vol. 22, Iss. 3, 2013-02 ,pp. : 558-567

Oxford University Press

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Mutations in the RP1 gene causing autosomal dominant retinitis pigmentosa

By Bowne Sara J.   Daiger Stephen P.   Hims Matthew M.  

Human Molecular Genetics, Vol. 8, Iss. 11, 1999-10 ,pp. : 2121-2128

Oxford University Press

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New mouse models for recessive retinitis pigmentosa caused by mutations in the Pde6a gene

By Sakamoto Kenji   McCluskey Michael   Wensel Theodore G.   Naggert Jrgen K.   Nishina Patsy M.  

Human Molecular Genetics, Vol. 18, Iss. 1, 2009-01 ,pp. : 178-192

Oxford University Press

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