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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. 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 retinitis pigmentosa caused by MFRP mutations: human phenotype and preliminary proof of concept.
Dinculescu A, Estreicher J, Zenteno JC, Aleman TS, Schwartz SB, Huang WC, Roman AJ, Sumaroka A, Li Q, Deng WT, Min SH, Chiodo VA, Neeley A, Liu X, Shu X, Matias-Florentino M, Buentello-Volante B, Boye SL, Cideciyan AV, Hauswirth WW, Jacobson SG. Dinculescu A, et al. Among authors: deng wt. Hum Gene Ther. 2012 Apr;23(4):367-76. doi: 10.1089/hum.2011.169. Epub 2012 Jan 26. Hum Gene Ther. 2012. PMID: 22142163 Free PMC article.
Gene therapy rescues photoreceptor blindness in dogs and paves the way for treating human X-linked retinitis pigmentosa.
Beltran WA, Cideciyan AV, Lewin AS, Iwabe S, Khanna H, Sumaroka A, Chiodo VA, Fajardo DS, Román AJ, Deng WT, Swider M, Alemán TS, Boye SL, Genini S, Swaroop A, Hauswirth WW, Jacobson SG, Aguirre GD. Beltran WA, et al. Among authors: deng wt. Proc Natl Acad Sci U S A. 2012 Feb 7;109(6):2132-7. doi: 10.1073/pnas.1118847109. Epub 2012 Jan 23. Proc Natl Acad Sci U S A. 2012. PMID: 22308428 Free PMC article.
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: deng wt. PLoS One. 2012;7(4):e35250. doi: 10.1371/journal.pone.0035250. Epub 2012 Apr 11. PLoS One. 2012. PMID: 22509403 Free PMC article.
Preclinical potency and safety studies of an AAV2-mediated gene therapy vector for the treatment of MERTK associated retinitis pigmentosa.
Conlon TJ, Deng WT, Erger K, Cossette T, Pang JJ, Ryals R, Clément N, Cleaver B, McDoom I, Boye SE, Peden MC, Sherwood MB, Abernathy CR, Alkuraya FS, Boye SL, Hauswirth WW. Conlon TJ, et al. Among authors: deng wt. Hum Gene Ther Clin Dev. 2013 Mar;24(1):23-8. doi: 10.1089/humc.2013.037. Epub 2013 Apr 3. Hum Gene Ther Clin Dev. 2013. PMID: 23692380 Free PMC article.
Cone phosphodiesterase-6α' restores rod function and confers distinct physiological properties in the rod phosphodiesterase-6β-deficient rd10 mouse.
Deng WT, Sakurai K, Kolandaivelu S, Kolesnikov AV, Dinculescu A, Li J, Zhu P, Liu X, Pang J, Chiodo VA, Boye SL, Chang B, Ramamurthy V, Kefalov VJ, Hauswirth WW. Deng WT, et al. J Neurosci. 2013 Jul 17;33(29):11745-53. doi: 10.1523/JNEUROSCI.1536-13.2013. J Neurosci. 2013. PMID: 23864662 Free PMC article.
54 results