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

M. H Tan, A. J Smith, B Pawlyk, X Xu, X Liu, J. B Bainbridge, M Basche, J McIntosh, H. V Tran, A Nathwani, T Li and R. R. Ali

Human Molecular Genetics 18 ( 12 ): 2099 - 2114 , 2009