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Page 1
LHON gene therapy vector prevents visual loss and optic neuropathy induced by G11778A mutant mitochondrial DNA: biodistribution and toxicology profile.
Koilkonda R, Yu H, Talla V, Porciatti V, Feuer WJ, Hauswirth WW, Chiodo V, Erger KE, Boye SL, Lewin AS, Conlon TJ, Renner L, Neuringer M, Detrisac C, Guy J. Koilkonda R, et al. Among authors: erger ke. Invest Ophthalmol Vis Sci. 2014 Oct 23;55(12):7739-53. doi: 10.1167/iovs.14-15388. Invest Ophthalmol Vis Sci. 2014. PMID: 25342621 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: erger k. 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.
Treatment of retinitis pigmentosa due to MERTK mutations by ocular subretinal injection of adeno-associated virus gene vector: results of a phase I trial.
Ghazi NG, Abboud EB, Nowilaty SR, Alkuraya H, Alhommadi A, Cai H, Hou R, Deng WT, Boye SL, Almaghamsi A, Al Saikhan F, Al-Dhibi H, Birch D, Chung C, Colak D, LaVail MM, Vollrath D, Erger K, Wang W, Conlon T, Zhang K, Hauswirth W, Alkuraya FS. Ghazi NG, et al. Among authors: erger k. Hum Genet. 2016 Mar;135(3):327-43. doi: 10.1007/s00439-016-1637-y. Epub 2016 Jan 29. Hum Genet. 2016. PMID: 26825853 Clinical Trial.
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. 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.
23 results