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Items: 1 to 20 of 36

1.

Prevention of autosomal dominant retinitis pigmentosa by systemic drug therapy targeting heat shock protein 90 (Hsp90).

Tam LC, Kiang AS, Campbell M, Keaney J, Farrar GJ, Humphries MM, Kenna PF, Humphries P.

Hum Mol Genet. 2010 Nov 15;19(22):4421-36. doi: 10.1093/hmg/ddq369. Epub 2010 Sep 2.

2.

Gene therapeutic approaches for dominant retinopathies.

Farrar GJ, Palfi A, O'Reilly M.

Curr Gene Ther. 2010 Oct;10(5):381-8. Review.

PMID:
20712579
3.

In vivo application of an RNAi strategy for the selective suppression of a mutant allele.

Kubodera T, Yamada H, Anzai M, Ohira S, Yokota S, Hirai Y, Mochizuki H, Shimada T, Mitani T, Mizusawa H, Yokota T.

Hum Gene Ther. 2011 Jan;22(1):27-34. doi: 10.1089/hum.2010.054.

PMID:
20649474
4.

Viral vector-mediated RNA interference.

Couto LB, High KA.

Curr Opin Pharmacol. 2010 Oct;10(5):534-42. doi: 10.1016/j.coph.2010.06.007. Epub 2010 Jul 9. Review.

PMID:
20620113
5.

Restoration of visual function in P23H rhodopsin transgenic rats by gene delivery of BiP/Grp78.

Gorbatyuk MS, Knox T, LaVail MM, Gorbatyuk OS, Noorwez SM, Hauswirth WW, Lin JH, Muzyczka N, Lewin AS.

Proc Natl Acad Sci U S A. 2010 Mar 30;107(13):5961-6. doi: 10.1073/pnas.0911991107. Epub 2010 Mar 15.

6.

Photoreceptor degeneration: genetic and mechanistic dissection of a complex trait.

Wright AF, Chakarova CF, Abd El-Aziz MM, Bhattacharya SS.

Nat Rev Genet. 2010 Apr;11(4):273-84. doi: 10.1038/nrg2717. Review.

PMID:
20212494
7.

Little vector, big gene transduction: fragmented genome reassembly of adeno-associated virus.

Hirsch ML, Agbandje-McKenna M, Samulski RJ.

Mol Ther. 2010 Jan;18(1):6-8. doi: 10.1038/mt.2009.280. No abstract available.

8.

Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration.

Simonelli F, Maguire AM, Testa F, Pierce EA, Mingozzi F, Bennicelli JL, Rossi S, Marshall K, Banfi S, Surace EM, Sun J, Redmond TM, Zhu X, Shindler KS, Ying GS, Ziviello C, Acerra C, Wright JF, McDonnell JW, High KA, Bennett J, Auricchio A.

Mol Ther. 2010 Mar;18(3):643-50. doi: 10.1038/mt.2009.277. Epub 2009 Dec 1.

9.

Evidence for the failure of adeno-associated virus serotype 5 to package a viral genome > or = 8.2 kb.

Lai Y, Yue Y, Duan D.

Mol Ther. 2010 Jan;18(1):75-9. doi: 10.1038/mt.2009.256. Epub 2009 Nov 10.

10.

Characterization of genome integrity for oversized recombinant AAV vector.

Dong B, Nakai H, Xiao W.

Mol Ther. 2010 Jan;18(1):87-92. doi: 10.1038/mt.2009.258. Epub 2009 Nov 10.

11.

Effect of genome size on AAV vector packaging.

Wu Z, Yang H, Colosi P.

Mol Ther. 2010 Jan;18(1):80-6. doi: 10.1038/mt.2009.255. Epub 2009 Nov 10.

12.

Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial.

Maguire AM, High KA, Auricchio A, Wright JF, Pierce EA, Testa F, Mingozzi F, Bennicelli JL, Ying GS, Rossi S, Fulton A, Marshall KA, Banfi S, Chung DC, Morgan JI, Hauck B, Zelenaia O, Zhu X, Raffini L, Coppieters F, De Baere E, Shindler KS, Volpe NJ, Surace EM, Acerra C, Lyubarsky A, Redmond TM, Stone E, Sun J, McDonnell JW, Leroy BP, Simonelli F, Bennett J.

Lancet. 2009 Nov 7;374(9701):1597-605. doi: 10.1016/S0140-6736(09)61836-5. Epub 2009 Oct 23. Erratum in: Lancet. 2010 Jan 2;375(9708):30.

13.

Adeno-associated virus-mediated rhodopsin replacement provides therapeutic benefit in mice with a targeted disruption of the rhodopsin gene.

Palfi A, Millington-Ward S, Chadderton N, O'Reilly M, Goldmann T, Humphries MM, Li T, Wolfrum U, Humphries P, Kenna PF, Farrar GJ.

Hum Gene Ther. 2010 Mar;21(3):311-23. doi: 10.1089/hum.2009.119.

PMID:
19824806
14.
15.

Functional cone rescue by RdCVF protein in a dominant model of retinitis pigmentosa.

Yang Y, Mohand-Said S, Danan A, Simonutti M, Fontaine V, Clerin E, Picaud S, LĂ©veillard T, Sahel JA.

Mol Ther. 2009 May;17(5):787-95. doi: 10.1038/mt.2009.28. Epub 2009 Mar 10.

16.

Improved retinal function in a mouse model of dominant retinitis pigmentosa following AAV-delivered gene therapy.

Chadderton N, Millington-Ward S, Palfi A, O'Reilly M, Tuohy G, Humphries MM, Li T, Humphries P, Kenna PF, Farrar GJ.

Mol Ther. 2009 Apr;17(4):593-9. doi: 10.1038/mt.2008.301. Epub 2009 Jan 27.

17.

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.

Hum Gene Ther. 2008 Oct;19(10):979-90. doi: 10.1089/hum.2008.107.

18.

Successful RPE65 gene replacement and improved visual function in humans.

Koenekoop RK.

Ophthalmic Genet. 2008 Sep;29(3):89-91. doi: 10.1080/13816810802216480.

PMID:
18766986
19.

Effect of gene therapy on visual function in Leber's congenital amaurosis.

Bainbridge JW, Smith AJ, Barker SS, Robbie S, Henderson R, Balaggan K, Viswanathan A, Holder GE, Stockman A, Tyler N, Petersen-Jones S, Bhattacharya SS, Thrasher AJ, Fitzke FW, Carter BJ, Rubin GS, Moore AT, Ali RR.

N Engl J Med. 2008 May 22;358(21):2231-9. doi: 10.1056/NEJMoa0802268. Epub 2008 Apr 27.

20.

Safety and efficacy of gene transfer for Leber's congenital amaurosis.

Maguire AM, Simonelli F, Pierce EA, Pugh EN Jr, Mingozzi F, Bennicelli J, Banfi S, Marshall KA, Testa F, Surace EM, Rossi S, Lyubarsky A, Arruda VR, Konkle B, Stone E, Sun J, Jacobs J, Dell'Osso L, Hertle R, Ma JX, Redmond TM, Zhu X, Hauck B, Zelenaia O, Shindler KS, Maguire MG, Wright JF, Volpe NJ, McDonnell JW, Auricchio A, High KA, Bennett J.

N Engl J Med. 2008 May 22;358(21):2240-8. doi: 10.1056/NEJMoa0802315. Epub 2008 Apr 27.

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