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REPEATABILITY AND LONGITUDINAL ASSESSMENT OF FOVEAL CONE STRUCTURE IN CNGB3-ASSOCIATED ACHROMATOPSIA.
Langlo CS, Erker LR, Parker M, Patterson EJ, Higgins BP, Summerfelt P, Razeen MM, Collison FT, Fishman GA, Kay CN, Zhang J, Weleber RG, Yang P, Pennesi ME, Lam BL, Chulay JD, Dubra A, Hauswirth WW, Wilson DJ, Carroll J; ACHM-001 study group. Langlo CS, et al. Among authors: hauswirth ww. Retina. 2017 Oct;37(10):1956-1966. doi: 10.1097/IAE.0000000000001434. Retina. 2017. PMID: 28145975 Free PMC article. Clinical Trial.
Restoration of cone vision in a mouse model of achromatopsia.
Alexander JJ, Umino Y, Everhart D, Chang B, Min SH, Li Q, Timmers AM, Hawes NL, Pang JJ, Barlow RB, Hauswirth WW. Alexander JJ, et al. Among authors: hauswirth ww. Nat Med. 2007 Jun;13(6):685-7. doi: 10.1038/nm1596. Epub 2007 May 21. Nat Med. 2007. PMID: 17515894 Free PMC article.
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.
Achromatopsia as a potential candidate for gene therapy.
Pang JJ, Alexander J, Lei B, Deng W, Zhang K, Li Q, Chang B, Hauswirth WW. Pang JJ, et al. Among authors: hauswirth ww. Adv Exp Med Biol. 2010;664:639-46. doi: 10.1007/978-1-4419-1399-9_73. Adv Exp Med Biol. 2010. PMID: 20238068 Free PMC article.
Gene therapy for leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 years.
Jacobson SG, Cideciyan AV, Ratnakaram R, Heon E, Schwartz SB, Roman AJ, Peden MC, Aleman TS, Boye SL, Sumaroka A, Conlon TJ, Calcedo R, Pang JJ, Erger KE, Olivares MB, Mullins CL, Swider M, Kaushal S, Feuer WJ, Iannaccone A, Fishman GA, Stone EM, Byrne BJ, Hauswirth WW. Jacobson SG, et al. Among authors: hauswirth ww. Arch Ophthalmol. 2012 Jan;130(1):9-24. doi: 10.1001/archophthalmol.2011.298. Epub 2011 Sep 12. Arch Ophthalmol. 2012. PMID: 21911650 Free PMC article. Clinical Trial.
AAV-mediated cone rescue in a naturally occurring mouse model of CNGA3-achromatopsia.
Pang JJ, Deng WT, Dai X, Lei B, Everhart D, Umino Y, Li J, Zhang K, Mao S, Boye SL, Liu L, Chiodo VA, Liu X, Shi W, Tao Y, Chang B, Hauswirth WW. Pang JJ, et al. Among authors: hauswirth ww. PLoS One. 2012;7(4):e35250. doi: 10.1371/journal.pone.0035250. Epub 2012 Apr 11. PLoS One. 2012. PMID: 22509403 Free PMC article.
Advancing therapeutic strategies for inherited retinal degeneration: recommendations from the Monaciano Symposium.
Thompson DA, Ali RR, Banin E, Branham KE, Flannery JG, Gamm DM, Hauswirth WW, Heckenlively JR, Iannaccone A, Jayasundera KT, Khan NW, Molday RS, Pennesi ME, Reh TA, Weleber RG, Zacks DN; Monaciano Consortium. Thompson DA, et al. Among authors: hauswirth ww. Invest Ophthalmol Vis Sci. 2015 Feb 9;56(2):918-31. doi: 10.1167/iovs.14-16049. Invest Ophthalmol Vis Sci. 2015. PMID: 25667399 Free PMC article. Review.
364 results