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Items: 1 to 20 of 41

1.

Microglial response to light-induced photoreceptor degeneration in the mouse retina.

Santos AM, Martín-Oliva D, Ferrer-Martín RM, Tassi M, Calvente R, Sierra A, Carrasco MC, Marín-Teva JL, Navascués J, Cuadros MA.

J Comp Neurol. 2010 Feb 15;518(4):477-92. doi: 10.1002/cne.22227.

PMID:
20020538
2.

Inner limiting membrane barriers to AAV-mediated retinal transduction from the vitreous.

Dalkara D, Kolstad KD, Caporale N, Visel M, Klimczak RR, Schaffer DV, Flannery JG.

Mol Ther. 2009 Dec;17(12):2096-102. doi: 10.1038/mt.2009.181. Epub 2009 Aug 11.

3.

RPE65: role in the visual cycle, human retinal disease, and gene therapy.

Cai X, Conley SM, Naash MI.

Ophthalmic Genet. 2009 Jun;30(2):57-62. doi: 10.1080/13816810802626399. Review.

4.

Connexin57 is expressed in dendro-dendritic and axo-axonal gap junctions of mouse horizontal cells and its distribution is modulated by light.

Janssen-Bienhold U, Trümpler J, Hilgen G, Schultz K, Müller LP, Sonntag S, Dedek K, Dirks P, Willecke K, Weiler R.

J Comp Neurol. 2009 Apr 1;513(4):363-74. doi: 10.1002/cne.21965.

PMID:
19177557
5.

High-efficiency transduction of the mouse retina by tyrosine-mutant AAV serotype vectors.

Petrs-Silva H, Dinculescu A, Li Q, Min SH, Chiodo V, Pang JJ, Zhong L, Zolotukhin S, Srivastava A, Lewin AS, Hauswirth WW.

Mol Ther. 2009 Mar;17(3):463-71. doi: 10.1038/mt.2008.269. Epub 2008 Dec 16.

6.

Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics.

Cideciyan AV, Aleman TS, Boye SL, Schwartz SB, Kaushal S, Roman AJ, Pang JJ, Sumaroka A, Windsor EA, Wilson JM, Flotte TR, Fishman GA, Heon E, Stone EM, Byrne BJ, Jacobson SG, Hauswirth WW.

Proc Natl Acad Sci U S A. 2008 Sep 30;105(39):15112-7. doi: 10.1073/pnas.0807027105. Epub 2008 Sep 22.

7.

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.

8.

Self-complementary AAV vectors; advances and applications.

McCarty DM.

Mol Ther. 2008 Oct;16(10):1648-56. doi: 10.1038/mt.2008.171. Epub 2008 Aug 5. Review.

9.

Next generation of adeno-associated virus 2 vectors: point mutations in tyrosines lead to high-efficiency transduction at lower doses.

Zhong L, Li B, Mah CS, Govindasamy L, Agbandje-McKenna M, Cooper M, Herzog RW, Zolotukhin I, Warrington KH Jr, Weigel-Van Aken KA, Hobbs JA, Zolotukhin S, Muzyczka N, Srivastava A.

Proc Natl Acad Sci U S A. 2008 Jun 3;105(22):7827-32. doi: 10.1073/pnas.0802866105. Epub 2008 May 29. Erratum in: Proc Natl Acad Sci U S A. 2008 Aug 5;105(31):11032.

10.

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.

11.

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.

12.

Novel AAV serotypes for improved ocular gene transfer.

Lebherz C, Maguire A, Tang W, Bennett J, Wilson JM.

J Gene Med. 2008 Apr;10(4):375-82. doi: 10.1002/jgm.1126.

13.

Reversal of blindness in animal models of leber congenital amaurosis using optimized AAV2-mediated gene transfer.

Bennicelli J, Wright JF, Komaromy A, Jacobs JB, Hauck B, Zelenaia O, Mingozzi F, Hui D, Chung D, Rex TS, Wei Z, Qu G, Zhou S, Zeiss C, Arruda VR, Acland GM, Dell'Osso LF, High KA, Maguire AM, Bennett J.

Mol Ther. 2008 Mar;16(3):458-65. doi: 10.1038/sj.mt.6300389. Epub 2008 Jan 22.

14.

Assessment of ocular transduction using single-stranded and self-complementary recombinant adeno-associated virus serotype 2/8.

Natkunarajah M, Trittibach P, McIntosh J, Duran Y, Barker SE, Smith AJ, Nathwani AC, Ali RR.

Gene Ther. 2008 Mar;15(6):463-7. Epub 2007 Nov 15.

PMID:
18004402
15.

Versatility of AAV vectors for retinal gene transfer.

Surace EM, Auricchio A.

Vision Res. 2008 Feb;48(3):353-9. Epub 2007 Oct 17. Review.

16.

AAV-mediated gene transfer for retinal diseases.

Allocca M, Tessitore A, Cotugno G, Auricchio A.

Expert Opin Biol Ther. 2006 Dec;6(12):1279-94. Review.

PMID:
17223737
17.

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.

Hum Gene Ther. 2006 Aug;17(8):845-58.

PMID:
16942444
18.

Recombinant adeno-associated virus serotype 9 leads to preferential cardiac transduction in vivo.

Pacak CA, Mah CS, Thattaliyath BD, Conlon TJ, Lewis MA, Cloutier DE, Zolotukhin I, Tarantal AF, Byrne BJ.

Circ Res. 2006 Aug 18;99(4):e3-9. Epub 2006 Jul 27.

19.

Robust systemic transduction with AAV9 vectors in mice: efficient global cardiac gene transfer superior to that of AAV8.

Inagaki K, Fuess S, Storm TA, Gibson GA, Mctiernan CF, Kay MA, Nakai H.

Mol Ther. 2006 Jul;14(1):45-53. Epub 2006 May 19.

20.

Long-term expression and repeated administration of AAV type 1, 2 and 5 vectors in skeletal muscle of immunocompetent adult mice.

Rivière C, Danos O, Douar AM.

Gene Ther. 2006 Sep;13(17):1300-8. Epub 2006 May 11.

PMID:
16688207

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