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Gene therapy provides long-term visual function in a pre-clinical model of retinitis pigmentosa.

Wert KJ, Davis RJ, Sancho-Pelluz J, Nishina PM, Tsang SH.

Hum Mol Genet. 2013 Feb 1;22(3):558-67. doi: 10.1093/hmg/dds466. Epub 2012 Oct 29.


Mid-stage intervention achieves similar efficacy as conventional early-stage treatment using gene therapy in a pre-clinical model of retinitis pigmentosa.

Wert KJ, Sancho-Pelluz J, Tsang SH.

Hum Mol Genet. 2014 Jan 15;23(2):514-23. doi: 10.1093/hmg/ddt452. Epub 2013 Sep 18.


Restoration of vision in the pde6β-deficient dog, a large animal model of rod-cone dystrophy.

Petit L, Lhériteau E, Weber M, Le Meur G, Deschamps JY, Provost N, Mendes-Madeira A, Libeau L, Guihal C, Colle MA, Moullier P, Rolling F.

Mol Ther. 2012 Nov;20(11):2019-30. doi: 10.1038/mt.2012.134. Epub 2012 Jul 24.


AAV delivery of wild-type rhodopsin preserves retinal function in a mouse model of autosomal dominant retinitis pigmentosa.

Mao H, James T Jr, Schwein A, Shabashvili AE, Hauswirth WW, Gorbatyuk MS, Lewin AS.

Hum Gene Ther. 2011 May;22(5):567-75. doi: 10.1089/hum.2010.140. Epub 2011 Mar 7.


AAV-mediated gene therapy for retinal degeneration in the rd10 mouse containing a recessive PDEbeta mutation.

Pang JJ, Boye SL, Kumar A, Dinculescu A, Deng W, Li J, Li Q, Rani A, Foster TC, Chang B, Hawes NL, Boatright JH, Hauswirth WW.

Invest Ophthalmol Vis Sci. 2008 Oct;49(10):4278-83. doi: 10.1167/iovs.07-1622. Epub 2008 Jun 27.


Therapeutic margins in a novel preclinical model of retinitis pigmentosa.

Davis RJ, Hsu CW, Tsai YT, Wert KJ, Sancho-Pelluz J, Lin CS, Tsang SH.

J Neurosci. 2013 Aug 14;33(33):13475-83. doi: 10.1523/JNEUROSCI.0419-13.2013.


Long-term retinal function and structure rescue using capsid mutant AAV8 vector in the rd10 mouse, a model of recessive retinitis pigmentosa.

Pang JJ, Dai X, Boye SE, Barone I, Boye SL, Mao S, Everhart D, Dinculescu A, Liu L, Umino Y, Lei B, Chang B, Barlow R, Strettoi E, Hauswirth WW.

Mol Ther. 2011 Feb;19(2):234-42. doi: 10.1038/mt.2010.273. Epub 2010 Dec 7.


Long-term protection of retinal structure but not function using RAAV.CNTF in animal models of retinitis pigmentosa.

Liang FQ, Aleman TS, Dejneka NS, Dudus L, Fisher KJ, Maguire AM, Jacobson SG, Bennett J.

Mol Ther. 2001 Nov;4(5):461-72.


Gene therapy for retinitis pigmentosa and Leber congenital amaurosis caused by defects in AIPL1: effective rescue of mouse models of partial and complete Aipl1 deficiency using AAV2/2 and AAV2/8 vectors.

Tan MH, Smith AJ, Pawlyk B, Xu X, Liu X, Bainbridge JB, Basche M, McIntosh J, Tran HV, Nathwani A, Li T, Ali RR.

Hum Mol Genet. 2009 Jun 15;18(12):2099-114. doi: 10.1093/hmg/ddp133. Epub 2009 Mar 19. Erratum in: Hum Mol Genet. 2010 Feb 15;19(4):735.


shRNA knockdown of guanylate cyclase 2e or cyclic nucleotide gated channel alpha 1 increases photoreceptor survival in a cGMP phosphodiesterase mouse model of retinitis pigmentosa.

Tosi J, Davis RJ, Wang NK, Naumann M, Lin CS, Tsang SH.

J Cell Mol Med. 2011 Aug;15(8):1778-87. doi: 10.1111/j.1582-4934.2010.01201.x.


Lentivirus-mediated expression of cDNA and shRNA slows degeneration in retinitis pigmentosa.

Tosi J, Sancho-Pelluz J, Davis RJ, Hsu CW, Wolpert KV, Sengillo JD, Lin CS, Tsang SH.

Exp Biol Med (Maywood). 2011 Oct;236(10):1211-7. doi: 10.1258/ebm.2011.011053. Epub 2011 Sep 1.


Long-term rescue of retinal structure and function by rhodopsin RNA replacement with a single adeno-associated viral vector in P23H RHO transgenic mice.

Mao H, Gorbatyuk MS, Rossmiller B, Hauswirth WW, Lewin AS.

Hum Gene Ther. 2012 Apr;23(4):356-66. doi: 10.1089/hum.2011.213. Epub 2012 Mar 28.


Gene therapy restores vision and delays degeneration in the CNGB1(-/-) mouse model of retinitis pigmentosa.

Koch S, Sothilingam V, Garcia Garrido M, Tanimoto N, Becirovic E, Koch F, Seide C, Beck SC, Seeliger MW, Biel M, Mühlfriedel R, Michalakis S.

Hum Mol Genet. 2012 Oct 15;21(20):4486-96. Epub 2012 Jul 16.


Mutation-independent rescue of a novel mouse model of Retinitis Pigmentosa.

Greenwald DL, Cashman SM, Kumar-Singh R.

Gene Ther. 2013 Apr;20(4):425-34. doi: 10.1038/gt.2012.53. Epub 2012 Jul 19.


Functional rescue of degenerating photoreceptors in mice homozygous for a hypomorphic cGMP phosphodiesterase 6 b allele (Pde6bH620Q).

Davis RJ, Tosi J, Janisch KM, Kasanuki JM, Wang NK, Kong J, Tsui I, Cilluffo M, Woodruff ML, Fain GL, Lin CS, Tsang SH.

Invest Ophthalmol Vis Sci. 2008 Nov;49(11):5067-76. doi: 10.1167/iovs.07-1422. Epub 2008 Jul 24.


Knockdown of wild-type mouse rhodopsin using an AAV vectored ribozyme as part of an RNA replacement approach.

Gorbatyuk MS, Pang JJ, Thomas J Jr, Hauswirth WW, Lewin AS.

Mol Vis. 2005 Aug 29;11:648-56.


Preservation of photoreceptor morphology and function in P23H rats using an allele independent ribozyme.

Gorbatyuk M, Justilien V, Liu J, Hauswirth WW, Lewin AS.

Exp Eye Res. 2007 Jan;84(1):44-52. Epub 2006 Nov 1.


Gene delivery of wild-type rhodopsin rescues retinal function in an autosomal dominant retinitis pigmentosa mouse model.

Mao H, Gorbatyuk MS, Hauswirth WW, Lewin AS.

Adv Exp Med Biol. 2012;723:199-205. doi: 10.1007/978-1-4614-0631-0_27. No abstract available.


Genetically modified neural stem cells for a local and sustained delivery of neuroprotective factors to the dystrophic mouse retina.

Jung G, Sun J, Petrowitz B, Riecken K, Kruszewski K, Jankowiak W, Kunst F, Skevas C, Richard G, Fehse B, Bartsch U.

Stem Cells Transl Med. 2013 Dec;2(12):1001-10. doi: 10.5966/sctm.2013-0013. Epub 2013 Oct 28.

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