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Long-Term Effects of Gene Therapy in a Novel Mouse Model of Human MFRP-Associated Retinopathy.
Chekuri A, Sahu B, Chavali VRM, Voronchikhina M, Soto-Hermida A, Suk JJ, Alapati AN, Bartsch DU, Ayala-Ramirez R, Zenteno JC, Dinculescu A, Jablonski MM, Borooah S, Ayyagari R. Chekuri A, et al. Among authors: dinculescu a. Hum Gene Ther. 2019 May;30(5):632-650. doi: 10.1089/hum.2018.192. Epub 2019 Jan 16. Hum Gene Ther. 2019. PMID: 30499344 Free PMC article.
Novel properties of tyrosine-mutant AAV2 vectors in the mouse retina.
Petrs-Silva H, Dinculescu A, Li Q, Deng WT, Pang JJ, Min SH, Chiodo V, Neeley AW, Govindasamy L, Bennett A, Agbandje-McKenna M, Zhong L, Li B, Jayandharan GR, Srivastava A, Lewin AS, Hauswirth WW. Petrs-Silva H, et al. Among authors: dinculescu a. Mol Ther. 2011 Feb;19(2):293-301. doi: 10.1038/mt.2010.234. Epub 2010 Nov 2. Mol Ther. 2011. PMID: 21045809 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. 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.
46 results