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Items: 1 to 50 of 137

1.

Adeno-associated virus vectored immunoprophylaxis to prevent HIV in healthy adults: a phase 1 randomised controlled trial.

Priddy FH, Lewis DJM, Gelderblom HC, Hassanin H, Streatfield C, LaBranche C, Hare J, Cox JH, Dally L, Bendel D, Montefiori D, Sayeed E, Ackland J, Gilmour J, Schnepp BC, Wright JF, Johnson P.

Lancet HIV. 2019 Apr;6(4):e230-e239. doi: 10.1016/S2352-3018(19)30003-7. Epub 2019 Mar 15. Erratum in: Lancet HIV. 2019 Apr 3;:.

2.

Retinal AAV8-RS1 Gene Therapy for X-Linked Retinoschisis: Initial Findings from a Phase I/IIa Trial by Intravitreal Delivery.

Cukras C, Wiley HE, Jeffrey BG, Sen HN, Turriff A, Zeng Y, Vijayasarathy C, Marangoni D, Ziccardi L, Kjellstrom S, Park TK, Hiriyanna S, Wright JF, Colosi P, Wu Z, Bush RA, Wei LL, Sieving PA.

Mol Ther. 2018 Sep 5;26(9):2282-2294. doi: 10.1016/j.ymthe.2018.05.025. Epub 2018 Jul 7.

PMID:
30196853
3.

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.

4.

Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial.

Russell S, Bennett J, Wellman JA, Chung DC, Yu ZF, Tillman A, Wittes J, Pappas J, Elci O, McCague S, Cross D, Marshall KA, Walshire J, Kehoe TL, Reichert H, Davis M, Raffini L, George LA, Hudson FP, Dingfield L, Zhu X, Haller JA, Sohn EH, Mahajan VB, Pfeifer W, Weckmann M, Johnson C, Gewaily D, Drack A, Stone E, Wachtel K, Simonelli F, Leroy BP, Wright JF, High KA, Maguire AM.

Lancet. 2017 Aug 26;390(10097):849-860. doi: 10.1016/S0140-6736(17)31868-8. Epub 2017 Jul 14. Erratum in: Lancet. 2017 Aug 26;390(10097):848.

5.

Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial.

Bennett J, Wellman J, Marshall KA, McCague S, Ashtari M, DiStefano-Pappas J, Elci OU, Chung DC, Sun J, Wright JF, Cross DR, Aravand P, Cyckowski LL, Bennicelli JL, Mingozzi F, Auricchio A, Pierce EA, Ruggiero J, Leroy BP, Simonelli F, High KA, Maguire AM.

Lancet. 2016 Aug 13;388(10045):661-72. doi: 10.1016/S0140-6736(16)30371-3. Epub 2016 Jun 30.

6.

Towards routine manufacturing of gene therapy drugs.

Merten OW, Wright JF.

Mol Ther Methods Clin Dev. 2016 Apr 13;3:16021. doi: 10.1038/mtm.2016.21. eCollection 2016. No abstract available.

7.

Effects of FVIII immunity on hepatocyte and hematopoietic stem cell-directed gene therapy of murine hemophilia A.

Lytle AM, Brown HC, Paik NY, Knight KA, Wright JF, Spencer HT, Doering CB.

Mol Ther Methods Clin Dev. 2016 Feb 10;3:15056. doi: 10.1038/mtm.2015.56. eCollection 2016.

8.

Debunking Hospice Myths.

Wright JF.

Home Healthc Now. 2016 Feb;34(2):108-9. doi: 10.1097/NHH.0000000000000336. No abstract available.

PMID:
26835813
9.

Progress and challenges in viral vector manufacturing.

van der Loo JC, Wright JF.

Hum Mol Genet. 2016 Apr 15;25(R1):R42-52. doi: 10.1093/hmg/ddv451. Epub 2015 Oct 30. Review.

10.

Differential Requirements for IL-17A and IL-22 in Cecal versus Colonic Inflammation Induced by Helicobacter hepaticus.

Morrison PJ, Ballantyne SJ, Macdonald SJ, Moore JW, Jenkins D, Wright JF, Fouser LA, Kullberg MC.

Am J Pathol. 2015 Dec;185(12):3290-303. doi: 10.1016/j.ajpath.2015.08.015. Epub 2015 Oct 14.

11.

Preclinical Potency and Biodistribution Studies of an AAV 5 Vector Expressing Human Interferon-β (ART-I02) for Local Treatment of Patients with Rheumatoid Arthritis.

Aalbers CJ, Bevaart L, Loiler S, de Cortie K, Wright JF, Mingozzi F, Tak PP, Vervoordeldonk MJ.

PLoS One. 2015 Jun 24;10(6):e0130612. doi: 10.1371/journal.pone.0130612. eCollection 2015.

12.

Safety, Biodistribution, and Efficacy of an AAV-5 Vector Encoding Human Interferon-Beta (ART-I02) Delivered via Intra-Articular Injection in Rhesus Monkeys with Collagen-Induced Arthritis.

Bevaart L, Aalbers CJ, Vierboom MP, Broekstra N, Kondova I, Breedveld E, Hauck B, Wright JF, Tak PP, Vervoordeldonk MJ.

Hum Gene Ther Clin Dev. 2015 Jun;26(2):103-12. doi: 10.1089/humc.2015.009.

PMID:
26086763
13.

SAFETY AND TOLERABILITY OF MRI-GUIDED INFUSION OF AAV2-hAADC INTO THE MID-BRAIN OF NON-HUMAN PRIMATE.

San Sebastian W, Kells AP, Bringas J, Samaranch L, Hadaczek P, Ciesielska A, Macayan M, Pivirotto PJ, Forsayeth J, Osborne S, Wright JF, Green F, Heller G, Bankiewicz KS.

Mol Ther Methods Clin Dev. 2014 Oct 15;3. pii: 14049.

14.

Manufacturing and characterization of a recombinant adeno-associated virus type 8 reference standard material.

Ayuso E, Blouin V, Lock M, McGorray S, Leon X, Alvira MR, Auricchio A, Bucher S, Chtarto A, Clark KR, Darmon C, Doria M, Fountain W, Gao G, Gao K, Giacca M, Kleinschmidt J, Leuchs B, Melas C, Mizukami H, Müller M, Noordman Y, Bockstael O, Ozawa K, Pythoud C, Sumaroka M, Surosky R, Tenenbaum L, van der Linden I, Weins B, Wright JF, Zhang X, Zentilin L, Bosch F, Snyder RO, Moullier P.

Hum Gene Ther. 2014 Nov;25(11):977-87. doi: 10.1089/hum.2014.057.

15.

Product-Related Impurities in Clinical-Grade Recombinant AAV Vectors: Characterization and Risk Assessment.

Wright JF.

Biomedicines. 2014 Mar 3;2(1):80-97. doi: 10.3390/biomedicines2010080. Review.

16.

AAV empty capsids: for better or for worse?

Wright JF.

Mol Ther. 2014 Jan;22(1):1-2. doi: 10.1038/mt.2013.268. No abstract available.

17.

Bioengineered coagulation factor VIII enables long-term correction of murine hemophilia A following liver-directed adeno-associated viral vector delivery.

Brown HC, Wright JF, Zhou S, Lytle AM, Shields JE, Spencer HT, Doering CB.

Mol Ther Methods Clin Dev. 2014 Aug 6;1:14036. doi: 10.1038/mtm.2014.36. eCollection 2014.

18.

Overcoming preexisting humoral immunity to AAV using capsid decoys.

Mingozzi F, Anguela XM, Pavani G, Chen Y, Davidson RJ, Hui DJ, Yazicioglu M, Elkouby L, Hinderer CJ, Faella A, Howard C, Tai A, Podsakoff GM, Zhou S, Basner-Tschakarjan E, Wright JF, High KA.

Sci Transl Med. 2013 Jul 17;5(194):194ra92. doi: 10.1126/scitranslmed.3005795.

19.

Advancing translational research through the NHLBI Gene Therapy Resource Program (GTRP).

McDonald CL, Benson J, Cornetta K, Diggins M, Johnston JC, Sepelak S, Wang G, Wilson JM, Wright JF, Skarlatos SI.

Hum Gene Ther Clin Dev. 2013 Mar;24(1):5-10. doi: 10.1089/humc.2013.036. Epub 2013 Apr 3. Review.

20.

Biological Insights into Therapeutic Protein Modifications throughout Trafficking and Their Biopharmaceutical Applications.

Zhong X, Wright JF.

Int J Cell Biol. 2013;2013:273086. doi: 10.1155/2013/273086. Epub 2013 Apr 18.

21.

Chimeric antigen receptor-modified T cells for acute lymphoid leukemia.

Grupp SA, Kalos M, Barrett D, Aplenc R, Porter DL, Rheingold SR, Teachey DT, Chew A, Hauck B, Wright JF, Milone MC, Levine BL, June CH.

N Engl J Med. 2013 Apr 18;368(16):1509-1518. doi: 10.1056/NEJMoa1215134. Epub 2013 Mar 25. Erratum in: N Engl J Med. 2016 Mar 10;374(10):998.

22.

AAV-mediated expression of an ADAMTS13 variant prevents shigatoxin-induced thrombotic thrombocytopenic purpura.

Jin SY, Xiao J, Bao J, Zhou S, Wright JF, Zheng XL.

Blood. 2013 May 9;121(19):3825-9, S1-3. doi: 10.1182/blood-2013-02-486779. Epub 2013 Mar 20.

23.

Three-year follow-up after unilateral subretinal delivery of adeno-associated virus in patients with Leber congenital Amaurosis type 2.

Testa F, Maguire AM, Rossi S, Pierce EA, Melillo P, Marshall K, Banfi S, Surace EM, Sun J, Acerra C, Wright JF, Wellman J, High KA, Auricchio A, Bennett J, Simonelli F.

Ophthalmology. 2013 Jun;120(6):1283-91. doi: 10.1016/j.ophtha.2012.11.048. Epub 2013 Mar 6.

24.

Th17-cell plasticity in Helicobacter hepaticus-induced intestinal inflammation.

Morrison PJ, Bending D, Fouser LA, Wright JF, Stockinger B, Cooke A, Kullberg MC.

Mucosal Immunol. 2013 Nov;6(6):1143-56. doi: 10.1038/mi.2013.11. Epub 2013 Mar 6.

PMID:
23462910
25.

Pyroglutamate and O-linked glycan determine functional production of anti-IL17A and anti-IL22 peptide-antibody bispecific genetic fusions.

Zhong X, Kieras E, Sousa E, D'Antona A, Baber JC, He T, Desharnais J, Wood L, Luxenberg D, Stahl M, Kriz R, Lin L, Somers W, Fitz LJ, Wright JF.

J Biol Chem. 2013 Jan 11;288(2):1409-19. doi: 10.1074/jbc.M112.417717. Epub 2012 Nov 26.

26.

Unexpected mucin-type O-glycosylation and host-specific N-glycosylation of human recombinant interleukin-17A expressed in a human kidney cell line.

Geoghegan KF, Song X, Hoth LR, Feng X, Shanker S, Quazi A, Luxenberg DP, Wright JF, Griffor MC.

Protein Expr Purif. 2013 Jan;87(1):27-34. doi: 10.1016/j.pep.2012.09.013. Epub 2012 Oct 13.

PMID:
23069765
27.

Cerebral infusion of AAV9 vector-encoding non-self proteins can elicit cell-mediated immune responses.

Ciesielska A, Hadaczek P, Mittermeyer G, Zhou S, Wright JF, Bankiewicz KS, Forsayeth J.

Mol Ther. 2013 Jan;21(1):158-66. doi: 10.1038/mt.2012.167. Epub 2012 Aug 28.

28.

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.

29.

Pharmacological modulation of humoral immunity in a nonhuman primate model of AAV gene transfer for hemophilia B.

Mingozzi F, Chen Y, Murphy SL, Edmonson SC, Tai A, Price SD, Metzger ME, Zhou S, Wright JF, Donahue RE, Dunbar CE, High KA.

Mol Ther. 2012 Jul;20(7):1410-6. doi: 10.1038/mt.2012.84. Epub 2012 May 8.

30.

Stimulation of TLR4 by recombinant HSP70 requires structural integrity of the HSP70 protein itself.

Luong M, Zhang Y, Chamberlain T, Zhou T, Wright JF, Dower K, Hall JP.

J Inflamm (Lond). 2012 Mar 26;9:11. doi: 10.1186/1476-9255-9-11.

31.

Systemic errors in quantitative polymerase chain reaction titration of self-complementary adeno-associated viral vectors and improved alternative methods.

Fagone P, Wright JF, Nathwani AC, Nienhuis AW, Davidoff AM, Gray JT.

Hum Gene Ther Methods. 2012 Feb;23(1):1-7. doi: 10.1089/hgtb.2011.104.

32.

AAV2 gene therapy readministration in three adults with congenital blindness.

Bennett J, Ashtari M, Wellman J, Marshall KA, Cyckowski LL, Chung DC, McCague S, Pierce EA, Chen Y, Bennicelli JL, Zhu X, Ying GS, Sun J, Wright JF, Auricchio A, Simonelli F, Shindler KS, Mingozzi F, High KA, Maguire AM.

Sci Transl Med. 2012 Feb 8;4(120):120ra15. doi: 10.1126/scitranslmed.3002865.

33.

In vitro characterization of the activity of PF-05095808, a novel biological agent for hepatitis C virus therapy.

Lavender H, Brady K, Burden F, Delpuech-Adams O, Denise H, Palmer A, Perkins H, Savic B, Scott S, Smith-Burchnell C, Troke P, Wright JF, Suhy D, Corbau R.

Antimicrob Agents Chemother. 2012 Mar;56(3):1364-75. doi: 10.1128/AAC.05357-11. Epub 2011 Dec 27.

34.

Adeno-associated viral vector manufacturing: keeping pace with accelerating clinical development.

Wright JF.

Hum Gene Ther. 2011 Aug;22(8):913-4. doi: 10.1089/hum.2011.2514. No abstract available.

PMID:
21859253
35.

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
36.

New adeno-associated virus strategies to support momentum in the clinic.

Wright JF.

Hum Gene Ther. 2011 May;22(5):519-21. doi: 10.1089/hum.2011.4080. No abstract available.

PMID:
21521072
37.

Chronic mucocutaneous candidiasis in humans with inborn errors of interleukin-17 immunity.

Puel A, Cypowyj S, Bustamante J, Wright JF, Liu L, Lim HK, Migaud M, Israel L, Chrabieh M, Audry M, Gumbleton M, Toulon A, Bodemer C, El-Baghdadi J, Whitters M, Paradis T, Brooks J, Collins M, Wolfman NM, Al-Muhsen S, Galicchio M, Abel L, Picard C, Casanova JL.

Science. 2011 Apr 1;332(6025):65-8. doi: 10.1126/science.1200439. Epub 2011 Feb 24.

38.

PEG-modulated column chromatography for purification of recombinant adeno-associated virus serotype 9.

Zhou J, Yang X, Wright JF, High KA, Couto L, Qu G.

J Virol Methods. 2011 Apr;173(1):99-107. doi: 10.1016/j.jviromet.2011.01.013. Epub 2011 Feb 3.

PMID:
21295608
39.

Safety of liver gene transfer following peripheral intravascular delivery of adeno-associated virus (AAV)-5 and AAV-6 in a large animal model.

Favaro P, Finn JD, Siner JI, Wright JF, High KA, Arruda VR.

Hum Gene Ther. 2011 Jul;22(7):843-52. doi: 10.1089/hum.2010.155. Epub 2011 Mar 8.

40.

Assessing the potential for AAV vector genotoxicity in a murine model.

Li H, Malani N, Hamilton SR, Schlachterman A, Bussadori G, Edmonson SE, Shah R, Arruda VR, Mingozzi F, Wright JF, Bushman FD, High KA.

Blood. 2011 Mar 24;117(12):3311-9. doi: 10.1182/blood-2010-08-302729. Epub 2010 Nov 24. Erratum in: Blood. 2011 Jun 16;117(24):6739.

41.

Complement C3a, CpG oligos, and DNA/C3a complex stimulate IFN-α production in a receptor for advanced glycation end product-dependent manner.

Ruan BH, Li X, Winkler AR, Cunningham KM, Kuai J, Greco RM, Nocka KH, Fitz LJ, Wright JF, Pittman DD, Tan XY, Paulsen JE, Lin LL, Winkler DG.

J Immunol. 2010 Oct 1;185(7):4213-22. doi: 10.4049/jimmunol.1000863. Epub 2010 Sep 3.

42.

Manufacturing and regulatory strategies for clinical AAV2-hRPE65.

Wright JF, Wellman J, High KA.

Curr Gene Ther. 2010 Oct;10(5):341-9. Review.

PMID:
20712582
43.

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.

44.

Safety of AAV factor IX peripheral transvenular gene delivery to muscle in hemophilia B dogs.

Haurigot V, Mingozzi F, Buchlis G, Hui DJ, Chen Y, Basner-Tschakarjan E, Arruda VR, Radu A, Franck HG, Wright JF, Zhou S, Stedman HH, Bellinger DA, Nichols TC, High KA.

Mol Ther. 2010 Jul;18(7):1318-29. doi: 10.1038/mt.2010.73. Epub 2010 Apr 27.

45.

Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B.

Arruda VR, Stedman HH, Haurigot V, Buchlis G, Baila S, Favaro P, Chen Y, Franck HG, Zhou S, Wright JF, Couto LB, Jiang H, Pierce GF, Bellinger DA, Mingozzi F, Nichols TC, High KA.

Blood. 2010 Jun 10;115(23):4678-88. doi: 10.1182/blood-2009-12-261156. Epub 2010 Mar 24.

46.

A fluorescent assay suitable for inhibitor screening and vanin tissue quantification.

Ruan BH, Cole DC, Wu P, Quazi A, Page K, Wright JF, Huang N, Stock JR, Nocka K, Aulabaugh A, Krykbaev R, Fitz LJ, Wolfman NM, Fleming ML.

Anal Biochem. 2010 Apr 15;399(2):284-92. doi: 10.1016/j.ab.2009.12.010. Epub 2009 Dec 16.

PMID:
20018163
47.

High AAV vector purity results in serotype- and tissue-independent enhancement of transduction efficiency.

Ayuso E, Mingozzi F, Montane J, Leon X, Anguela XM, Haurigot V, Edmonson SA, Africa L, Zhou S, High KA, Bosch F, Wright JF.

Gene Ther. 2010 Apr;17(4):503-10. doi: 10.1038/gt.2009.157. Epub 2009 Dec 3.

PMID:
19956269
48.

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.

49.

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.

50.

Safety and tolerability of putaminal AADC gene therapy for Parkinson disease.

Christine CW, Starr PA, Larson PS, Eberling JL, Jagust WJ, Hawkins RA, VanBrocklin HF, Wright JF, Bankiewicz KS, Aminoff MJ.

Neurology. 2009 Nov 17;73(20):1662-9. doi: 10.1212/WNL.0b013e3181c29356. Epub 2009 Oct 14.

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