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Items: 1 to 50 of 335

1.

Dual ABCA4-AAV vector treatment reduces pathogenic retinal A2E accumulation in a mouse model of autosomal recessive Stargardt Disease.

Dyka FM, Molday LL, Chiodo V, Molday RS, Hauswirth WW.

Hum Gene Ther. 2019 Aug 16. doi: 10.1089/hum.2019.132. [Epub ahead of print]

PMID:
31418294
2.

Effects of a combinatorial treatment with gene and cell therapy on retinal ganglion cell survival and axonal outgrowth after optic nerve injury.

Nascimento-Dos-Santos G, Teixeira-Pinheiro LC, da Silva-Júnior AJ, Carvalho LRP, Mesentier-Louro LA, Hauswirth WW, Mendez-Otero R, Santiago MF, Petrs-Silva H.

Gene Ther. 2019 Jun 26. doi: 10.1038/s41434-019-0089-0. [Epub ahead of print]

PMID:
31243393
3.

Dual AAV-mediated gene therapy restores hearing in a DFNB9 mouse model.

Akil O, Dyka F, Calvet C, Emptoz A, Lahlou G, Nouaille S, Boutet de Monvel J, Hardelin JP, Hauswirth WW, Avan P, Petit C, Safieddine S, Lustig LR.

Proc Natl Acad Sci U S A. 2019 Feb 19. pii: 201817537. doi: 10.1073/pnas.1817537116. [Epub ahead of print]

4.

Rescue of cone function in cone-only Nphp5 knockout mouse model with Leber congenital amaurosis phenotype.

Hanke-Gogokhia C, Chiodo VA, Hauswirth WW, Frederick JM, Baehr W.

Mol Vis. 2018 Dec 30;24:834-846. eCollection 2018.

5.

Toxicology and Pharmacology of an AAV Vector Expressing Codon-Optimized RPGR in RPGR-Deficient Rd9 Mice.

Song C, Conlon TJ, Deng WT, Coleman KE, Zhu P, Plummer C, Mandapati S, Van Hoosear M, Green KB, Sonnentag P, Sharma AK, Timmers A, Robinson PM, Knop DR, Hauswirth WW, Chulay JD, Shearman MS, Ye GJ.

Hum Gene Ther Clin Dev. 2018 Dec;29(4):188-197. doi: 10.1089/humc.2018.168.

PMID:
30280954
6.

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
7.

Mutation-independent rhodopsin gene therapy by knockdown and replacement with a single AAV vector.

Cideciyan AV, Sudharsan R, Dufour VL, Massengill MT, Iwabe S, Swider M, Lisi B, Sumaroka A, Marinho LF, Appelbaum T, Rossmiller B, Hauswirth WW, Jacobson SG, Lewin AS, Aguirre GD, Beltran WA.

Proc Natl Acad Sci U S A. 2018 Sep 4;115(36):E8547-E8556. doi: 10.1073/pnas.1805055115. Epub 2018 Aug 20.

8.

Cone Phosphodiesterase-6γ' Subunit Augments Cone PDE6 Holoenzyme Assembly and Stability in a Mouse Model Lacking Both Rod and Cone PDE6 Catalytic Subunits.

Deng WT, Kolandaivelu S, Dinculescu A, Li J, Zhu P, Chiodo VA, Ramamurthy V, Hauswirth WW.

Front Mol Neurosci. 2018 Jul 9;11:233. doi: 10.3389/fnmol.2018.00233. eCollection 2018.

9.

Six Years and Counting: Restoration of Photopic Retinal Function and Visual Behavior Following Gene Augmentation Therapy in a Sheep Model of CNGA3 Achromatopsia.

Ofri R, Averbukh E, Ezra-Elia R, Ross M, Honig H, Obolensky A, Rosov A, Hauswirth WW, Gootwine E, Banin E.

Hum Gene Ther. 2018 Jul 30. doi: 10.1089/hum.2018.076. [Epub ahead of print]

PMID:
29926749
10.

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
11.

Co-Expression of Wild-Type and Mutant S163R C1QTNF5 in Retinal Pigment Epithelium.

Dinculescu A, Dyka FM, Min SH, Stupay RM, Hooper MJ, Smith WC, Hauswirth WW.

Adv Exp Med Biol. 2018;1074:61-66. doi: 10.1007/978-3-319-75402-4_8.

PMID:
29721928
12.

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.

13.

BEST1 gene therapy corrects a diffuse retina-wide microdetachment modulated by light exposure.

Guziewicz KE, Cideciyan AV, Beltran WA, Komáromy AM, Dufour VL, Swider M, Iwabe S, Sumaroka A, Kendrick BT, Ruthel G, Chiodo VA, Héon E, Hauswirth WW, Jacobson SG, Aguirre GD.

Proc Natl Acad Sci U S A. 2018 Mar 20;115(12):E2839-E2848. doi: 10.1073/pnas.1720662115. Epub 2018 Mar 5.

14.

Reply.

Guy J, Feuer WJ, Davis JL, Porciatti V, Gonzalez PJ, Koilkonda RD, Yuan H, Hauswirth WW, Lam BL.

Ophthalmology. 2018 Feb;125(2):e15-e16. doi: 10.1016/j.ophtha.2017.08.041. No abstract available.

PMID:
29389414
15.

Human L- and M-opsins restore M-cone function in a mouse model for human blue cone monochromacy.

Deng WT, Li J, Zhu P, Chiodo VA, Smith WC, Freedman B, Baehr W, Pang J, Hauswirth WW.

Mol Vis. 2018 Jan 8;24:17-28. eCollection 2018.

16.

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.

17.

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.

18.

Safety and Efficacy of AAV5 Vectors Expressing Human or Canine CNGB3 in CNGB3-Mutant Dogs.

Ye GJ, Komáromy AM, Zeiss C, Calcedo R, Harman CD, Koehl KL, Stewart GA, Iwabe S, Chiodo VA, Hauswirth WW, Aguirre GD, Chulay JD.

Hum Gene Ther Clin Dev. 2017 Dec;28(4):197-207. doi: 10.1089/humc.2017.125. Epub 2017 Oct 11.

19.

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.

20.

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.

21.

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.

22.

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.

23.

Gene Therapy for Leber Hereditary Optic Neuropathy: Low- and Medium-Dose Visual Results.

Guy J, Feuer WJ, Davis JL, Porciatti V, Gonzalez PJ, Koilkonda RD, Yuan H, Hauswirth WW, Lam BL.

Ophthalmology. 2017 Nov;124(11):1621-1634. doi: 10.1016/j.ophtha.2017.05.016. Epub 2017 Jun 21.

24.

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
25.

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.

26.

Overexpression of the X-Linked Inhibitor of Apoptosis Protects Against Retinal Degeneration in a Feline Model of Retinal Detachment.

Wassmer SJ, Leonard BC, Coupland SG, Baker AN, Hamilton J, Hauswirth WW, Tsilfidis C.

Hum Gene Ther. 2017 Jun;28(6):482-492. doi: 10.1089/hum.2016.161. Epub 2017 Mar 23.

27.

Gene Augmentation Therapy for a Missense Substitution in the cGMP-Binding Domain of Ovine CNGA3 Gene Restores Vision in Day-Blind Sheep.

Gootwine E, Abu-Siam M, Obolensky A, Rosov A, Honig H, Nitzan T, Shirak A, Ezra-Elia R, Yamin E, Banin E, Averbukh E, Hauswirth WW, Ofri R, Seroussi E.

Invest Ophthalmol Vis Sci. 2017 Mar 1;58(3):1577-1584. doi: 10.1167/iovs.16-20986.

28.

rAAV8-733-Mediated Gene Transfer of CHIP/Stub-1 Prevents Hippocampal Neuronal Death in Experimental Brain Ischemia.

Cabral-Miranda F, Nicoloso-Simões E, Adão-Novaes J, Chiodo V, Hauswirth WW, Linden R, Chiarini LB, Petrs-Silva H.

Mol Ther. 2017 Feb 1;25(2):392-400. doi: 10.1016/j.ymthe.2016.11.017. Epub 2016 Dec 31.

29.

REPEATABILITY AND LONGITUDINAL ASSESSMENT OF FOVEAL CONE STRUCTURE IN CNGB3-ASSOCIATED ACHROMATOPSIA.

Langlo CS, Erker LR, Parker M, Patterson EJ, Higgins BP, Summerfelt P, Razeen MM, Collison FT, Fishman GA, Kay CN, Zhang J, Weleber RG, Yang P, Pennesi ME, Lam BL, Chulay JD, Dubra A, Hauswirth WW, Wilson DJ, Carroll J; ACHM-001 study group.

Retina. 2017 Oct;37(10):1956-1966. doi: 10.1097/IAE.0000000000001434.

30.

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.

31.

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.

32.

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.

33.

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.

34.

Characterization of intravitreally delivered capsid mutant AAV2-Cre vector to induce tissue-specific mutations in murine retinal ganglion cells.

Langouet-Astrie CJ, Yang Z, Polisetti SM, Welsbie DS, Hauswirth WW, Zack DJ, Merbs SL, Enke RA.

Exp Eye Res. 2016 Oct;151:61-7. doi: 10.1016/j.exer.2016.07.019. Epub 2016 Jul 30.

PMID:
27481653
35.

Residual Foveal Cone Structure in CNGB3-Associated Achromatopsia.

Langlo CS, Patterson EJ, Higgins BP, Summerfelt P, Razeen MM, Erker LR, Parker M, Collison FT, Fishman GA, Kay CN, Zhang J, Weleber RG, Yang P, Wilson DJ, Pennesi ME, Lam BL, Chiang J, Chulay JD, Dubra A, Hauswirth WW, Carroll J; ACHM-001 Study Group.

Invest Ophthalmol Vis Sci. 2016 Aug 1;57(10):3984-95. doi: 10.1167/iovs.16-19313.

36.

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.

37.

Occludin S490 Phosphorylation Regulates Vascular Endothelial Growth Factor-Induced Retinal Neovascularization.

Liu X, Dreffs A, Díaz-Coránguez M, Runkle EA, Gardner TW, Chiodo VA, Hauswirth WW, Antonetti DA.

Am J Pathol. 2016 Sep;186(9):2486-99. doi: 10.1016/j.ajpath.2016.04.018. Epub 2016 Jul 14.

38.

Results at 2 Years after Gene Therapy for RPE65-Deficient Leber Congenital Amaurosis and Severe Early-Childhood-Onset Retinal Dystrophy.

Weleber RG, Pennesi ME, Wilson DJ, Kaushal S, Erker LR, Jensen L, McBride MT, Flotte TR, Humphries M, Calcedo R, Hauswirth WW, Chulay JD, Stout JT.

Ophthalmology. 2016 Jul;123(7):1606-20. doi: 10.1016/j.ophtha.2016.03.003. Epub 2016 Apr 19.

39.

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
40.

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.

41.

Safety and Biodistribution Evaluation in Cynomolgus Macaques of rAAV2tYF-PR1.7-hCNGB3, a Recombinant AAV Vector for Treatment of Achromatopsia.

Ye GJ, Budzynski E, Sonnentag P, Nork TM, Miller PE, Sharma AK, Ver Hoeve JN, Smith LM, Arndt T, Calcedo R, Gaskin C, Robinson PM, Knop DR, Hauswirth WW, Chulay JD.

Hum Gene Ther Clin Dev. 2016 Mar;27(1):37-48. doi: 10.1089/humc.2015.164.

42.

Safety and Biodistribution Evaluation in CNGB3-Deficient Mice of rAAV2tYF-PR1.7-hCNGB3, a Recombinant AAV Vector for Treatment of Achromatopsia.

Ye GJ, Budzynski E, Sonnentag P, Nork TM, Miller PE, McPherson L, Ver Hoeve JN, Smith LM, Arndt T, Mandapati S, Robinson PM, Calcedo R, Knop DR, Hauswirth WW, Chulay JD.

Hum Gene Ther Clin Dev. 2016 Mar;27(1):27-36. doi: 10.1089/humc.2015.163.

43.

Maintaining ocular safety with light exposure, focusing on devices for optogenetic stimulation.

Yan B, Vakulenko M, Min SH, Hauswirth WW, Nirenberg S.

Vision Res. 2016 Apr;121:57-71. doi: 10.1016/j.visres.2016.01.006. Epub 2016 Feb 26.

44.

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.

45.

Targeting the Nrf2 Signaling Pathway in the Retina With a Gene-Delivered Secretable and Cell-Penetrating Peptide.

Ildefonso CJ, Jaime H, Brown EE, Iwata RL, Ahmed CM, Massengill MT, Biswal MR, Boye SE, Hauswirth WW, Ash JD, Li Q, Lewin AS.

Invest Ophthalmol Vis Sci. 2016 Feb;57(2):372-86. doi: 10.1167/iovs.15-17703.

46.

Healthy and diseased corticospinal motor neurons are selectively transduced upon direct AAV2-2 injection into the motor cortex.

Jara JH, Stanford MJ, Zhu Y, Tu M, Hauswirth WW, Bohn MC, DeVries SH, Özdinler PH.

Gene Ther. 2016 Mar;23(3):272-82. doi: 10.1038/gt.2015.112. Epub 2016 Jan 21.

47.

Adeno-Associated Virus Type 2 and Hepatocellular Carcinoma?

Berns KI, Byrne BJ, Flotte TR, Gao G, Hauswirth WW, Herzog RW, Muzyczka N, VandenDriessche T, Xiao X, Zolotukhin S, Srivastava A.

Hum Gene Ther. 2015 Dec;26(12):779-81. doi: 10.1089/hum.2015.29014.kib. No abstract available.

48.

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.

49.

Pathological Effects of Mutant C1QTNF5 (S163R) Expression in Murine Retinal Pigment Epithelium.

Dinculescu A, Min SH, Dyka FM, Deng WT, Stupay RM, Chiodo V, Smith WC, Hauswirth WW.

Invest Ophthalmol Vis Sci. 2015 Oct;56(11):6971-80. doi: 10.1167/iovs.15-17166.

50.

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.

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