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Items: 1 to 20 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.

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