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Items: 17

1.

Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant.

George LA, Sullivan SK, Giermasz A, Rasko JEJ, Samelson-Jones BJ, Ducore J, Cuker A, Sullivan LM, Majumdar S, Teitel J, McGuinn CE, Ragni MV, Luk AY, Hui D, Wright JF, Chen Y, Liu Y, Wachtel K, Winters A, Tiefenbacher S, Arruda VR, van der Loo JCM, Zelenaia O, Takefman D, Carr ME, Couto LB, Anguela XM, High KA.

N Engl J Med. 2017 Dec 7;377(23):2215-2227. doi: 10.1056/NEJMoa1708538.

2.

Safe, long-term hepatic expression of anti-HCV shRNA in a nonhuman primate model.

Suhy DA, Kao SC, Mao T, Whiteley L, Denise H, Souberbielle B, Burdick AD, Hayes K, Wright JF, Lavender H, Roelvink P, Kolykhalov A, Brady K, Moschos SA, Hauck B, Zelenaia O, Zhou S, Scribner C, High KA, Renison SH, Corbau R.

Mol Ther. 2012 Sep;20(9):1737-49. doi: 10.1038/mt.2012.119. Epub 2012 Jun 26.

3.

Vector characterization methods for quality control testing of recombinant adeno-associated viruses.

Wright JF, Zelenaia O.

Methods Mol Biol. 2011;737:247-78. doi: 10.1007/978-1-61779-095-9_11.

PMID:
21590401
4.

Characterization of a recombinant adeno-associated virus type 2 Reference Standard Material.

Lock M, McGorray S, Auricchio A, Ayuso E, Beecham EJ, Blouin-Tavel V, Bosch F, Bose M, Byrne BJ, Caton T, Chiorini JA, Chtarto A, Clark KR, Conlon T, Darmon C, Doria M, Douar A, Flotte TR, Francis JD, Francois A, Giacca M, Korn MT, Korytov I, Leon X, Leuchs B, Lux G, Melas C, Mizukami H, Moullier P, Müller M, Ozawa K, Philipsberg T, Poulard K, Raupp C, Rivière C, Roosendaal SD, Samulski RJ, Soltys SM, Surosky R, Tenenbaum L, Thomas DL, van Montfort B, Veres G, Wright JF, Xu Y, Zelenaia O, Zentilin L, Snyder RO.

Hum Gene Ther. 2010 Oct;21(10):1273-85. doi: 10.1089/hum.2009.223.

5.

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.

6.

Undetectable transcription of cap in a clinical AAV vector: implications for preformed capsid in immune responses.

Hauck B, Murphy SL, Smith PH, Qu G, Liu X, Zelenaia O, Mingozzi F, Sommer JM, High KA, Wright JF.

Mol Ther. 2009 Jan;17(1):144-52. doi: 10.1038/mt.2008.227. Epub 2008 Oct 21.

7.

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.

8.

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.

9.

Regulation of astrocytic glutamate transporter expression by Akt: evidence for a selective transcriptional effect on the GLT-1/EAAT2 subtype.

Li LB, Toan SV, Zelenaia O, Watson DJ, Wolfe JH, Rothstein JD, Robinson MB.

J Neurochem. 2006 May;97(3):759-71. Epub 2006 Mar 29.

10.

Evidence that Akt mediates platelet-derived growth factor-dependent increases in activity and surface expression of the neuronal glutamate transporter, EAAC1.

Krizman-Genda E, González MI, Zelenaia O, Robinson MB.

Neuropharmacology. 2005 Nov;49(6):872-82. Epub 2005 Sep 21.

PMID:
16182322
11.

Differential regulation of GLAST immunoreactivity and activity by protein kinase C: evidence for modification of amino and carboxyl termini.

Susarla BT, Seal RP, Zelenaia O, Watson DJ, Wolfe JH, Amara SG, Robinson MB.

J Neurochem. 2004 Dec;91(5):1151-63.

12.
13.

Epidermal growth factor receptor agonists increase expression of glutamate transporter GLT-1 in astrocytes through pathways dependent on phosphatidylinositol 3-kinase and transcription factor NF-kappaB.

Zelenaia O, Schlag BD, Gochenauer GE, Ganel R, Song W, Beesley JS, Grinspan JB, Rothstein JD, Robinson MB.

Mol Pharmacol. 2000 Apr;57(4):667-78.

PMID:
10727511
14.

Expression of the GLT-1 subtype of Na+-dependent glutamate transporter: pharmacological characterization and lack of regulation by protein kinase C.

Tan J, Zelenaia O, Correale D, Rothstein JD, Robinson MB.

J Pharmacol Exp Ther. 1999 Jun;289(3):1600-10.

PMID:
10336558
15.

Regulation of the glial Na+-dependent glutamate transporters by cyclic AMP analogs and neurons.

Schlag BD, Vondrasek JR, Munir M, Kalandadze A, Zelenaia OA, Rothstein JD, Robinson MB.

Mol Pharmacol. 1998 Mar;53(3):355-69.

PMID:
9495799
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