@article {504106,
	title = {AAV-mediated RLBP1 gene therapy improves the rate of dark adaptation in Rlbp1 knockout mice.},
	journal = {Mol Ther Methods Clin Dev},
	volume = {2},
	year = {2015},
	month = {2015},
	pages = {15022},
	abstract = {Recessive mutations in RLBP1 cause a form of retinitis pigmentosa in which the retina, before its degeneration leads to blindness, abnormally slowly recovers sensitivity after exposure to light. To develop a potential gene therapy for this condition, we tested multiple recombinant adeno-associated vectors (rAAVs) composed of different promoters, capsid serotypes, and genome conformations. We generated rAAVs in which sequences from the promoters of the human RLBP1, RPE65, or BEST1 genes drove the expression of a reporter gene (green fluorescent protein). A promoter derived from the RLBP1 gene mediated expression in the retinal pigment epithelium and M{\"u}ller cells (the intended target cell types) at qualitatively higher levels than in other retinal cell types in wild-type mice and monkeys. With this promoter upstream of the coding sequence of the human RLBP1 gene, we compared the potencies of vectors with an AAV2 versus an AAV8 capsid in transducing mouse retinas, and we compared vectors with a self-complementary versus a single-stranded genome. The optimal vector (scAAV8-pRLBP1-hRLBP1) had serotype 8 capsid and a self-complementary genome. Subretinal injection of scAAV8-pRLBP1-hRLBP1 in Rlbp1 nullizygous mice improved the rate of dark adaptation based on scotopic (rod-plus-cone) and photopic (cone) electroretinograms (ERGs). The effect was still present after 1 year.},
	issn = {2329-0501},
	doi = {10.1038/mtm.2015.22},
	author = {Choi, Vivian W and Bigelow, Chad E and McGee, Terri L and Gujar, Akshata N and Li, Hui and Hanks, Shawn M and Vrouvlianis, Joanna and Maker, Michael and Leehy, Barrett and Zhang, Yiqin and Aranda, Jorge and Bounoutas, George and Demirs, John T and Yang, Junzheng and Ornberg, Richard and Wang, Yu and Martin, Wendy and Stout, Kelly R and Argentieri, Gregory and Grosenstein, Paul and Diaz, Danielle and Turner, Oliver and Jaffee, Bruce D and Police, Seshidhar R and Dryja, Thaddeus P}
}
