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Page 1
Natural history of cone disease in the murine model of Leber congenital amaurosis due to CEP290 mutation: determining the timing and expectation of therapy.
Boye SE, Huang WC, Roman AJ, Sumaroka A, Boye SL, Ryals RC, Olivares MB, Ruan Q, Tucker BA, Stone EM, Swaroop A, Cideciyan AV, Hauswirth WW, Jacobson SG. Boye SE, et al. Among authors: hauswirth ww. PLoS One. 2014 Mar 26;9(3):e92928. doi: 10.1371/journal.pone.0092928. eCollection 2014. PLoS One. 2014. PMID: 24671090 Free PMC article.
Gene therapy restores vision in a canine model of childhood blindness.
Acland GM, Aguirre GD, Ray J, Zhang Q, Aleman TS, Cideciyan AV, Pearce-Kelling SE, Anand V, Zeng Y, Maguire AM, Jacobson SG, Hauswirth WW, Bennett J. Acland GM, et al. Among authors: hauswirth ww. Nat Genet. 2001 May;28(1):92-5. doi: 10.1038/ng0501-92. Nat Genet. 2001. PMID: 11326284
Gene therapy restores vision-dependent behavior as well as retinal structure and function in a mouse model of RPE65 Leber congenital amaurosis.
Pang JJ, Chang B, Kumar A, Nusinowitz S, Noorwez SM, Li J, Rani A, Foster TC, Chiodo VA, Doyle T, Li H, Malhotra R, Teusner JT, McDowell JH, Min SH, Li Q, Kaushal S, Hauswirth WW. Pang JJ, et al. Among authors: hauswirth ww. Mol Ther. 2006 Mar;13(3):565-72. doi: 10.1016/j.ymthe.2005.09.001. Epub 2005 Oct 11. Mol Ther. 2006. PMID: 16223604 Free article.
Long-term restoration of rod and cone vision by single dose rAAV-mediated gene transfer to the retina in a canine model of childhood blindness.
Acland GM, Aguirre GD, Bennett J, Aleman TS, Cideciyan AV, Bennicelli J, Dejneka NS, Pearce-Kelling SE, Maguire AM, Palczewski K, Hauswirth WW, Jacobson SG. Acland GM, et al. Among authors: hauswirth ww. Mol Ther. 2005 Dec;12(6):1072-82. doi: 10.1016/j.ymthe.2005.08.008. Epub 2005 Oct 14. Mol Ther. 2005. PMID: 16226919 Free PMC article.
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: hauswirth ww. 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.
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: hauswirth ww. Hum Gene Ther. 2006 Aug;17(8):845-58. doi: 10.1089/hum.2006.17.845. Hum Gene Ther. 2006. PMID: 16942444
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.
365 results