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Safety in nonhuman primates of ocular AAV2-RPE65, a candidate treatment for blindness in Leber congenital amaurosis.
Jacobson SG, Boye SL, Aleman TS, Conlon TJ, Zeiss CJ, Roman AJ, Cideciyan AV, Schwartz SB, Komaromy AM, Doobrajh M, Cheung AY, Sumaroka A, Pearce-Kelling SE, Aguirre GD, Kaushal S, Maguire AM, Flotte TR, Hauswirth WW. Jacobson SG, et al. Among authors: conlon tj. Hum Gene Ther. 2006 Aug;17(8):845-58. doi: 10.1089/hum.2006.17.845. Hum Gene Ther. 2006. PMID: 16942444
Safety of recombinant adeno-associated virus type 2-RPE65 vector delivered by ocular subretinal injection.
Jacobson SG, Acland GM, Aguirre GD, Aleman TS, Schwartz SB, Cideciyan AV, Zeiss CJ, Komaromy AM, Kaushal S, Roman AJ, Windsor EA, Sumaroka A, Pearce-Kelling SE, Conlon TJ, Chiodo VA, Boye SL, Flotte TR, Maguire AM, Bennett J, Hauswirth WW. Jacobson SG, et al. Among authors: conlon tj. Mol Ther. 2006 Jun;13(6):1074-84. doi: 10.1016/j.ymthe.2006.03.005. Epub 2006 Apr 27. Mol Ther. 2006. PMID: 16644289 Free article.
Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial.
Hauswirth WW, Aleman TS, Kaushal S, Cideciyan AV, Schwartz SB, Wang L, Conlon TJ, Boye SL, Flotte TR, Byrne BJ, Jacobson SG. Hauswirth WW, et al. Among authors: conlon tj. Hum Gene Ther. 2008 Oct;19(10):979-90. doi: 10.1089/hum.2008.107. Hum Gene Ther. 2008. PMID: 18774912 Free PMC article. 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. Among authors: conlon tj. 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.
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. 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.
66 results