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Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics.
Cideciyan AV, Aleman TS, Boye SL, Schwartz SB, Kaushal S, Roman AJ, Pang JJ, Sumaroka A, Windsor EA, Wilson JM, Flotte TR, Fishman GA, Heon E, Stone EM, Byrne BJ, Jacobson SG, Hauswirth WW. Cideciyan AV, et al. Among authors: hauswirth ww. Proc Natl Acad Sci U S A. 2008 Sep 30;105(39):15112-7. doi: 10.1073/pnas.0807027105. Epub 2008 Sep 22. Proc Natl Acad Sci U S A. 2008. PMID: 18809924 Free PMC article. Clinical Trial.
Gene therapy restores vision in a canine model of childhood blindness.
Acland GM, Aguirre GD, Ray J, Zhang Q, Aleman TS, Cideciyan AV, Pearce-Kelling SE, Anand V, Zeng Y, Maguire AM, Jacobson SG, Hauswirth WW, Bennett J. Acland GM, et al. Among authors: hauswirth ww. Nat Genet. 2001 May;28(1):92-5. doi: 10.1038/ng0501-92. Nat Genet. 2001. PMID: 11326284
Interleukin 10 attenuates neointimal proliferation and inflammation in aortic allografts by a heme oxygenase-dependent pathway.
Chen S, Kapturczak MH, Wasserfall C, Glushakova OY, Campbell-Thompson M, Deshane JS, Joseph R, Cruz PE, Hauswirth WW, Madsen KM, Croker BP, Berns KI, Atkinson MA, Flotte TR, Tisher CC, Agarwal A. Chen S, et al. Among authors: hauswirth ww. Proc Natl Acad Sci U S A. 2005 May 17;102(20):7251-6. doi: 10.1073/pnas.0502407102. Epub 2005 May 6. Proc Natl Acad Sci U S A. 2005. PMID: 15878989 Free PMC article.
Gene therapy restores vision-dependent behavior as well as retinal structure and function in a mouse model of RPE65 Leber congenital amaurosis.
Pang JJ, Chang B, Kumar A, Nusinowitz S, Noorwez SM, Li J, Rani A, Foster TC, Chiodo VA, Doyle T, Li H, Malhotra R, Teusner JT, McDowell JH, Min SH, Li Q, Kaushal S, Hauswirth WW. Pang JJ, et al. Among authors: hauswirth ww. Mol Ther. 2006 Mar;13(3):565-72. doi: 10.1016/j.ymthe.2005.09.001. Epub 2005 Oct 11. Mol Ther. 2006. PMID: 16223604 Free article.
Long-term restoration of rod and cone vision by single dose rAAV-mediated gene transfer to the retina in a canine model of childhood blindness.
Acland GM, Aguirre GD, Bennett J, Aleman TS, Cideciyan AV, Bennicelli J, Dejneka NS, Pearce-Kelling SE, Maguire AM, Palczewski K, Hauswirth WW, Jacobson SG. Acland GM, et al. Among authors: hauswirth ww. Mol Ther. 2005 Dec;12(6):1072-82. doi: 10.1016/j.ymthe.2005.08.008. Epub 2005 Oct 14. Mol Ther. 2005. PMID: 16226919 Free PMC article.
364 results