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Items: 1 to 50 of 100

1.

Impaired ABCA1/ABCG1-mediated lipid efflux in the mouse retinal pigment epithelium (RPE) leads to retinal degeneration.

Storti F, Klee K, Todorova V, Steiner R, Othman A, van der Velde-Visser S, Samardzija M, Meneau I, Barben M, Karademir D, Pauzuolyte V, Boye SL, Blaser F, Ullmer C, Dunaief JL, Hornemann T, Rohrer L, den Hollander A, von Eckardstein A, Fingerle J, Maugeais C, Grimm C.

Elife. 2019 Mar 13;8. pii: e45100. doi: 10.7554/eLife.45100.

2.

SubILM Injection of AAV for Gene Delivery to the Retina.

Gamlin PD, Alexander JJ, Boye SL, Witherspoon CD, Boye SE.

Methods Mol Biol. 2019;1950:249-262. doi: 10.1007/978-1-4939-9139-6_14.

3.

Somatic Gene Editing of GUCY2D by AAV-CRISPR/Cas9 Alters Retinal Structure and Function in Mouse and Macaque.

McCullough KT, Boye SL, Fajardo D, Calabro K, Peterson JJ, Strang CE, Chakraborty D, Gloskowski S, Haskett S, Samuelsson S, Jiang H, Witherspoon CD, Gamlin PD, Maeder ML, Boye SE.

Hum Gene Ther. 2019 May;30(5):571-589. doi: 10.1089/hum.2018.193. Epub 2018 Dec 20.

PMID:
30358434
4.

Complement C3-Targeted Gene Therapy Restricts Onset and Progression of Neurodegeneration in Chronic Mouse Glaucoma.

Bosco A, Anderson SR, Breen KT, Romero CO, Steele MR, Chiodo VA, Boye SL, Hauswirth WW, Tomlinson S, Vetter ML.

Mol Ther. 2018 Oct 3;26(10):2379-2396. doi: 10.1016/j.ymthe.2018.08.017. Epub 2018 Aug 24.

PMID:
30217731
5.

A Drug-Tunable Gene Therapy for Broad-Spectrum Protection against Retinal Degeneration.

Santiago CP, Keuthan CJ, Boye SL, Boye SE, Imam AA, Ash JD.

Mol Ther. 2018 Oct 3;26(10):2407-2417. doi: 10.1016/j.ymthe.2018.07.016. Epub 2018 Jul 19.

PMID:
30078764
6.

Overexpression of Type 3 Iodothyronine Deiodinase Reduces Cone Death in the Leber Congenital Amaurosis Model Mice.

Yang F, Ma H, Boye SL, Hauswirth WW, Ding XQ.

Adv Exp Med Biol. 2018;1074:125-131. doi: 10.1007/978-3-319-75402-4_16.

PMID:
29721936
7.

Publisher Correction: Gene-based Therapy in a Mouse Model of Blue Cone Monochromacy.

Zhang Y, Deng WT, Du W, Zhu P, Li J, Xu F, Sun J, Gerstner CD, Baehr W, Boye SL, Zhao C, Hauswirth WW, Pang JJ.

Sci Rep. 2018 Mar 14;8(1):4807. doi: 10.1038/s41598-018-23131-w.

8.

ELOVL4-Mediated Production of Very Long-Chain Ceramides Stabilizes Tight Junctions and Prevents Diabetes-Induced Retinal Vascular Permeability.

Kady NM, Liu X, Lydic TA, Syed MH, Navitskaya S, Wang Q, Hammer SS, O'Reilly S, Huang C, Seregin SS, Amalfitano A, Chiodo VA, Boye SL, Hauswirth WW, Antonetti DA, Busik JV.

Diabetes. 2018 Apr;67(4):769-781. doi: 10.2337/db17-1034. Epub 2018 Jan 23.

9.

Patients and animal models of CNGβ1-deficient retinitis pigmentosa support gene augmentation approach.

Petersen-Jones SM, Occelli LM, Winkler PA, Lee W, Sparrow JR, Tsukikawa M, Boye SL, Chiodo V, Capasso JE, Becirovic E, Schön C, Seeliger MW, Levin AV, Michalakis S, Hauswirth WW, Tsang SH.

J Clin Invest. 2018 Jan 2;128(1):190-206. doi: 10.1172/JCI95161. Epub 2017 Nov 20.

10.

Long-term photoreceptor rescue in two rodent models of retinitis pigmentosa by adeno-associated virus delivery of Stanniocalcin-1.

Roddy GW, Yasumura D, Matthes MT, Alavi MV, Boye SL, Rosa RH Jr, Fautsch MP, Hauswirth WW, LaVail MM.

Exp Eye Res. 2017 Dec;165:175-181. doi: 10.1016/j.exer.2017.09.011. Epub 2017 Sep 30.

11.

Rationally Engineered AAV Capsids Improve Transduction and Volumetric Spread in the CNS.

Kanaan NM, Sellnow RC, Boye SL, Coberly B, Bennett A, Agbandje-McKenna M, Sortwell CE, Hauswirth WW, Boye SE, Manfredsson FP.

Mol Ther Nucleic Acids. 2017 Sep 15;8:184-197. doi: 10.1016/j.omtn.2017.06.011. Epub 2017 Jun 21.

12.

Gene-based Therapy in a Mouse Model of Blue Cone Monochromacy.

Zhang Y, Deng WT, Du W, Zhu P, Li J, Xu F, Sun J, Gerstner CD, Baehr W, Boye SL, Zhao C, Hauswirth WW, Pang JJ.

Sci Rep. 2017 Jul 27;7(1):6690. doi: 10.1038/s41598-017-06982-7. Erratum in: Sci Rep. 2018 Mar 14;8(1):4807.

13.

Gene Therapy in a Large Animal Model of PDE6A-Retinitis Pigmentosa.

Mowat FM, Occelli LM, Bartoe JT, Gervais KJ, Bruewer AR, Querubin J, Dinculescu A, Boye SL, Hauswirth WW, Petersen-Jones SM.

Front Neurosci. 2017 Jun 20;11:342. doi: 10.3389/fnins.2017.00342. eCollection 2017.

14.

miRNA-mediated post-transcriptional silencing of transgenes leads to increased adeno-associated viral vector yield and targeting specificity.

Reid CA, Boye SL, Hauswirth WW, Lipinski DM.

Gene Ther. 2017 Aug;24(8):462-469. doi: 10.1038/gt.2017.50. Epub 2017 Jun 15.

PMID:
28617420
15.

Optimization of Retinal Gene Therapy for X-Linked Retinitis Pigmentosa Due to RPGR Mutations.

Beltran WA, Cideciyan AV, Boye SE, Ye GJ, Iwabe S, Dufour VL, Marinho LF, Swider M, Kosyk MS, Sha J, Boye SL, Peterson JJ, Witherspoon CD, Alexander JJ, Ying GS, Shearman MS, Chulay JD, Hauswirth WW, Gamlin PD, Jacobson SG, Aguirre GD.

Mol Ther. 2017 Aug 2;25(8):1866-1880. doi: 10.1016/j.ymthe.2017.05.004. Epub 2017 May 27.

16.

Efficient Gene Delivery and Expression in Pancreas and Pancreatic Tumors by Capsid-Optimized AAV8 Vectors.

Chen M, Maeng K, Nawab A, Francois RA, Bray JK, Reinhard MK, Boye SL, Hauswirth WW, Kaye FJ, Aslanidi G, Srivastava A, Zajac-Kaye M.

Hum Gene Ther Methods. 2017 Feb;28(1):49-59. doi: 10.1089/hgtb.2016.089.

17.

Novel Methodology for Creating Macaque Retinas with Sortable Photoreceptors and Ganglion Cells.

Choudhury S, Strang CE, Alexander JJ, Scalabrino ML, Lynch Hill J, Kasuga DT, Witherspoon CD, Boye SL, Gamlin PD, Boye SE.

Front Neurosci. 2016 Dec 1;10:551. eCollection 2016.

18.

Functional study of two biochemically unusual mutations in GUCY2D Leber congenital amaurosis expressed via adenoassociated virus vector in mouse retinas.

Boye SL, Olshevskaya EV, Peshenko IV, McCullough KT, Boye SE, Dizhoor AM.

Mol Vis. 2016 Nov 10;22:1342-1351. eCollection 2016.

19.

NF1 Is a Direct G Protein Effector Essential for Opioid Signaling to Ras in the Striatum.

Xie K, Colgan LA, Dao MT, Muntean BS, Sutton LP, Orlandi C, Boye SL, Boye SE, Shih CC, Li Y, Xu B, Smith RG, Yasuda R, Martemyanov KA.

Curr Biol. 2016 Nov 21;26(22):2992-3003. doi: 10.1016/j.cub.2016.09.010. Epub 2016 Oct 20.

20.

Targeting iodothyronine deiodinases locally in the retina is a therapeutic strategy for retinal degeneration.

Yang F, Ma H, Belcher J, Butler MR, Redmond TM, Boye SL, Hauswirth WW, Ding XQ.

FASEB J. 2016 Dec;30(12):4313-4325. Epub 2016 Sep 13.

21.

PAX6 MiniPromoters drive restricted expression from rAAV in the adult mouse retina.

Hickmott JW, Chen CY, Arenillas DJ, Korecki AJ, Lam SL, Molday LL, Bonaguro RJ, Zhou M, Chou AY, Mathelier A, Boye SL, Hauswirth WW, Molday RS, Wasserman WW, Simpson EM.

Mol Ther Methods Clin Dev. 2016 Aug 10;3:16051. doi: 10.1038/mtm.2016.51. eCollection 2016.

22.

Small GTPases Rab8a and Rab11a Are Dispensable for Rhodopsin Transport in Mouse Photoreceptors.

Ying G, Gerstner CD, Frederick JM, Boye SL, Hauswirth WW, Baehr W.

PLoS One. 2016 Aug 16;11(8):e0161236. doi: 10.1371/journal.pone.0161236. eCollection 2016.

23.

Highly Efficient Delivery of Adeno-Associated Viral Vectors to the Primate Retina.

Boye SE, Alexander JJ, Witherspoon CD, Boye SL, Peterson JJ, Clark ME, Sandefer KJ, Girkin CA, Hauswirth WW, Gamlin PD.

Hum Gene Ther. 2016 Aug;27(8):580-97. doi: 10.1089/hum.2016.085.

24.

Photoreceptor-targeted gene delivery using intravitreally administered AAV vectors in dogs.

Boyd RF, Sledge DG, Boye SL, Boye SE, Hauswirth WW, Komáromy AM, Petersen-Jones SM, Bartoe JT.

Gene Ther. 2016 Apr;23(4):400. doi: 10.1038/gt.2016.10. No abstract available.

PMID:
27052928
25.

Reduced retinal transduction and enhanced transgene-directed immunogenicity with intravitreal delivery of rAAV following posterior vitrectomy in dogs.

Boyd RF, Boye SL, Conlon TJ, Erger KE, Sledge DG, Langohr IM, Hauswirth WW, Komáromy AM, Boye SE, Petersen-Jones SM, Bartoe JT.

Gene Ther. 2016 Jun;23(6):548-56. doi: 10.1038/gt.2016.31. Epub 2016 Apr 7.

26.

AAV-Mediated Clarin-1 Expression in the Mouse Retina: Implications for USH3A Gene Therapy.

Dinculescu A, Stupay RM, Deng WT, Dyka FM, Min SH, Boye SL, Chiodo VA, Abrahan CE, Zhu P, Li Q, Strettoi E, Novelli E, Nagel-Wolfrum K, Wolfrum U, Smith WC, Hauswirth WW.

PLoS One. 2016 Feb 16;11(2):e0148874. doi: 10.1371/journal.pone.0148874. eCollection 2016.

27.

Impact of Heparan Sulfate Binding on Transduction of Retina by Recombinant Adeno-Associated Virus Vectors.

Boye SL, Bennett A, Scalabrino ML, McCullough KT, Van Vliet K, Choudhury S, Ruan Q, Peterson J, Agbandje-McKenna M, Boye SE.

J Virol. 2016 Mar 28;90(8):4215-4231. doi: 10.1128/JVI.00200-16. Print 2016 Apr.

28.

Treatment of retinitis pigmentosa due to MERTK mutations by ocular subretinal injection of adeno-associated virus gene vector: results of a phase I trial.

Ghazi NG, Abboud EB, Nowilaty SR, Alkuraya H, Alhommadi A, Cai H, Hou R, Deng WT, Boye SL, Almaghamsi A, Al Saikhan F, Al-Dhibi H, Birch D, Chung C, Colak D, LaVail MM, Vollrath D, Erger K, Wang W, Conlon T, Zhang K, Hauswirth W, Alkuraya FS.

Hum Genet. 2016 Mar;135(3):327-43. doi: 10.1007/s00439-016-1637-y. Epub 2016 Jan 29.

PMID:
26825853
29.

Cone-Specific Promoters for Gene Therapy of Achromatopsia and Other Retinal Diseases.

Ye GJ, Budzynski E, Sonnentag P, Nork TM, Sheibani N, Gurel Z, Boye SL, Peterson JJ, Boye SE, Hauswirth WW, Chulay JD.

Hum Gene Ther. 2016 Jan;27(1):72-82. doi: 10.1089/hum.2015.130.

30.

Photoreceptor-targeted gene delivery using intravitreally administered AAV vectors in dogs.

Boyd RF, Sledge DG, Boye SL, Boye SE, Hauswirth WW, Komáromy AM, Petersen-Jones SM, Bartoe JT.

Gene Ther. 2016 Feb;23(2):223-30. doi: 10.1038/gt.2015.96. Epub 2015 Oct 15. Erratum in: Gene Ther. 2016 Apr;23(4):400.

31.

Successful arrest of photoreceptor and vision loss expands the therapeutic window of retinal gene therapy to later stages of disease.

Beltran WA, Cideciyan AV, Iwabe S, Swider M, Kosyk MS, McDaid K, Martynyuk I, Ying GS, Shaffer J, Deng WT, Boye SL, Lewin AS, Hauswirth WW, Jacobson SG, Aguirre GD.

Proc Natl Acad Sci U S A. 2015 Oct 27;112(43):E5844-53. doi: 10.1073/pnas.1509914112. Epub 2015 Oct 12.

32.

Consequences of zygote injection and germline transfer of mutant human mitochondrial DNA in mice.

Yu H, Koilkonda RD, Chou TH, Porciatti V, Mehta A, Hentall ID, Chiodo VA, Boye SL, Hauswirth WW, Lewin AS, Guy J.

Proc Natl Acad Sci U S A. 2015 Oct 20;112(42):E5689-98. doi: 10.1073/pnas.1506129112. Epub 2015 Oct 5.

33.

Systemic Vascular Transduction by Capsid Mutant Adeno-Associated Virus After Intravenous Injection.

Lipinski DM, Reid CA, Boye SL, Peterson JJ, Qi X, Boye SE, Boulton ME, Hauswirth WW.

Hum Gene Ther. 2015 Nov;26(11):767-76. doi: 10.1089/hum.2015.097. Epub 2015 Sep 29.

34.

Intravitreal delivery of a novel AAV vector targets ON bipolar cells and restores visual function in a mouse model of complete congenital stationary night blindness.

Scalabrino ML, Boye SL, Fransen KM, Noel JM, Dyka FM, Min SH, Ruan Q, De Leeuw CN, Simpson EM, Gregg RG, McCall MA, Peachey NS, Boye SE.

Hum Mol Genet. 2015 Nov 1;24(21):6229-39. doi: 10.1093/hmg/ddv341. Epub 2015 Aug 26.

35.

Gene Therapy Fully Restores Vision to the All-Cone Nrl(-/-) Gucy2e(-/-) Mouse Model of Leber Congenital Amaurosis-1.

Boye SL, Peterson JJ, Choudhury S, Min SH, Ruan Q, McCullough KT, Zhang Z, Olshevskaya EV, Peshenko IV, Hauswirth WW, Ding XQ, Dizhoor AM, Boye SE.

Hum Gene Ther. 2015 Sep;26(9):575-92. doi: 10.1089/hum.2015.053. Epub 2015 Aug 6.

36.

Stability and Safety of an AAV Vector for Treating RPGR-ORF15 X-Linked Retinitis Pigmentosa.

Deng WT, Dyka FM, Dinculescu A, Li J, Zhu P, Chiodo VA, Boye SL, Conlon TJ, Erger K, Cossette T, Hauswirth WW.

Hum Gene Ther. 2015 Sep;26(9):593-602. doi: 10.1089/hum.2015.035. Epub 2015 Jul 29.

37.

Capsid Mutated Adeno-Associated Virus Delivered to the Anterior Chamber Results in Efficient Transduction of Trabecular Meshwork in Mouse and Rat.

Bogner B, Boye SL, Min SH, Peterson JJ, Ruan Q, Zhang Z, Reitsamer HA, Hauswirth WW, Boye SE.

PLoS One. 2015 Jun 8;10(6):e0128759. doi: 10.1371/journal.pone.0128759. eCollection 2015.

38.

AAV8(Y733F)-mediated gene therapy in a Spata7 knockout mouse model of Leber congenital amaurosis and retinitis pigmentosa.

Zhong H, Eblimit A, Moayedi Y, Boye SL, Chiodo VA, Chen Y, Li Y, Nichols RM, Hauswirth WW, Chen R, Mardon G.

Gene Ther. 2015 Aug;22(8):619-27. doi: 10.1038/gt.2015.42. Epub 2015 May 12.

39.

Complex I subunit gene therapy with NDUFA6 ameliorates neurodegeneration in EAE.

Talla V, Koilkonda R, Porciatti V, Chiodo V, Boye SL, Hauswirth WW, Guy J.

Invest Ophthalmol Vis Sci. 2015 Jan 22;56(2):1129-40. doi: 10.1167/iovs.14-15950.

40.

Targeted gene delivery to the enteric nervous system using AAV: a comparison across serotypes and capsid mutants.

Benskey MJ, Kuhn NC, Galligan JJ, Garcia J, Boye SE, Hauswirth WW, Mueller C, Boye SL, Manfredsson FP.

Mol Ther. 2015 Mar;23(3):488-500. doi: 10.1038/mt.2015.7. Epub 2015 Jan 16.

41.

LHON gene therapy vector prevents visual loss and optic neuropathy induced by G11778A mutant mitochondrial DNA: biodistribution and toxicology profile.

Koilkonda R, Yu H, Talla V, Porciatti V, Feuer WJ, Hauswirth WW, Chiodo V, Erger KE, Boye SL, Lewin AS, Conlon TJ, Renner L, Neuringer M, Detrisac C, Guy J.

Invest Ophthalmol Vis Sci. 2014 Oct 23;55(12):7739-53. doi: 10.1167/iovs.14-15388.

42.

Viral-mediated vision rescue of a novel AIPL1 cone-rod dystrophy model.

Ku CA, Chiodo VA, Boye SL, Hayes A, Goldberg AF, Hauswirth WW, Ramamurthy V.

Hum Mol Genet. 2015 Feb 1;24(3):670-84. doi: 10.1093/hmg/ddu487. Epub 2014 Sep 30.

43.

Gene therapy with mitochondrial heat shock protein 70 suppresses visual loss and optic atrophy in experimental autoimmune encephalomyelitis.

Talla V, Porciatti V, Chiodo V, Boye SL, Hauswirth WW, Guy J.

Invest Ophthalmol Vis Sci. 2014 Jul 11;55(8):5214-26. doi: 10.1167/iovs.14-14688.

44.

Targeted CNS Delivery Using Human MiniPromoters and Demonstrated Compatibility with Adeno-Associated Viral Vectors.

de Leeuw CN, Dyka FM, Boye SL, Laprise S, Zhou M, Chou AY, Borretta L, McInerny SC, Banks KG, Portales-Casamar E, Swanson MI, D'Souza CA, Boye SE, Jones SJ, Holt RA, Goldowitz D, Hauswirth WW, Wasserman WW, Simpson EM.

Mol Ther Methods Clin Dev. 2014 Jan 8;1:5.

45.

Inhibitor of apoptosis-stimulating protein of p53 (iASPP) is required for neuronal survival after axonal injury.

Wilson AM, Chiodo VA, Boye SL, Brecha NC, Hauswirth WW, Di Polo A.

PLoS One. 2014 Apr 8;9(4):e94175. doi: 10.1371/journal.pone.0094175. eCollection 2014.

46.

Natural history of cone disease in the murine model of Leber congenital amaurosis due to CEP290 mutation: determining the timing and expectation of therapy.

Boye SE, Huang WC, Roman AJ, Sumaroka A, Boye SL, Ryals RC, Olivares MB, Ruan Q, Tucker BA, Stone EM, Swaroop A, Cideciyan AV, Hauswirth WW, Jacobson SG.

PLoS One. 2014 Mar 26;9(3):e92928. doi: 10.1371/journal.pone.0092928. eCollection 2014.

47.

Cone specific promoter for use in gene therapy of retinal degenerative diseases.

Dyka FM, Boye SL, Ryals RC, Chiodo VA, Boye SE, Hauswirth WW.

Adv Exp Med Biol. 2014;801:695-701. doi: 10.1007/978-1-4614-3209-8_87.

48.

Dual adeno-associated virus vectors result in efficient in vitro and in vivo expression of an oversized gene, MYO7A.

Dyka FM, Boye SL, Chiodo VA, Hauswirth WW, Boye SE.

Hum Gene Ther Methods. 2014 Apr;25(2):166-77. doi: 10.1089/hgtb.2013.212.

49.

Safety and effects of the vector for the Leber hereditary optic neuropathy gene therapy clinical trial.

Koilkonda RD, Yu H, Chou TH, Feuer WJ, Ruggeri M, Porciatti V, Tse D, Hauswirth WW, Chiodo V, Boye SL, Lewin AS, Neuringer M, Renner L, Guy J.

JAMA Ophthalmol. 2014 Apr 1;132(4):409-20. doi: 10.1001/jamaophthalmol.2013.7630.

50.

Tyrosine capsid-mutant AAV vectors for gene delivery to the canine retina from a subretinal or intravitreal approach.

Mowat FM, Gornik KR, Dinculescu A, Boye SL, Hauswirth WW, Petersen-Jones SM, Bartoe JT.

Gene Ther. 2014 Jan;21(1):96-105. doi: 10.1038/gt.2013.64. Epub 2013 Nov 14.

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