Search Results - "Stargardt disease"

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  1. 1
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    Molecular diagnosis of putative Stargardt disease probands by exome sequencing by Strom Samuel P, Gao Yong-Qing, Martinez Ariadna, Ortube Carolina, Chen Zugen, Nelson Stanley F, Nusinowitz Steven, Farber Deborah B, Gorin Michael B

    Published 2012
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  2. 2
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    Novel variants of ABCA4 in Han Chinese families with Stargardt disease by Fang-Yuan Hu, Feng-Juan Gao, Jian-kang Li, Ping Xu, Dan-Dan Wang, Sheng-Hai Zhang, Ji-Hong Wu

    Published 2020
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  3. 3
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    Detailed Phenotyping and Therapeutic Strategies for Intronic ABCA4 Variants in Stargardt Disease by Mubeen Khan, Gavin Arno, Ana Fakin, David A. Parfitt, Patty P.A. Dhooge, Silvia Albert, Nathalie M. Bax, Lonneke Duijkers, Michael Niblock, Kwan L. Hau, Edward Bloch, Elena R. Schiff, Davide Piccolo, Michael C. Hogden, Carel B. Hoyng, Andrew R. Webster, Frans P.M. Cremers, Michael E. Cheetham, Alejandro Garanto, Rob W.J. Collin

    Published 2020
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  4. 4
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    Effective gene therapy of Stargardt disease with PEG-ECO/pGRK1-ABCA4-S/MAR nanoparticles by Da Sun, Wenyu Sun, Song-Qi Gao, Jonathan Lehrer, Amirreza Naderi, Cheng Wei, Sangjoon Lee, Andrew L. Schilb, Josef Scheidt, Ryan C. Hall, Elias I. Traboulsi, Krzysztof Palczewski, Zheng-Rong Lu

    Published 2022
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  5. 5
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    Efficient correction of ABCA4 variants by CRISPR-Cas9 in hiPSCs derived from Stargardt disease patients by Laura Siles, Sheila Ruiz-Nogales, Arnau Navinés-Ferrer, Pilar Méndez-Vendrell, Esther Pomares

    Published 2023
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  6. 6
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    Antisense oligonucleotide therapy corrects splicing in the common Stargardt disease type 1-causing variant ABCA4 c.5461-10T>C by Melita Kaltak, Petra de Bruijn, Davide Piccolo, Sang-Eun Lee, Kalyan Dulla, Thomas Hoogenboezem, Wouter Beumer, Andrew R. Webster, Rob W.J. Collin, Michael E. Cheetham, Gerard Platenburg, Jim Swildens

    Published 2023
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  7. 7
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    Penetration, distribution, and elimination of remofuscin/soraprazan in Stargardt mouse eyes following a single intravitreal injection using pharmacokinetics and transmission electron microscopic autoradiography: Implication for the local treatment of Stargardt's disease and dry age‐related macular degeneration by Sylvie Julien‐Schraermeyer, Barbara Illing, Alexander Tschulakow, Tatjana Taubitz, Jamil Guezguez, Michael Burnet, Ulrich Schraermeyer

    Published 2020
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  8. 8
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    Application of Retinal and Optic Nerve Imaging in Clinical Medicine 2.0

    Published 2023
    Subjects: “…Stargardt disease…”
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  9. 9
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    Molecular Therapies for Inherited Retinal Diseases

    Published 2020
    Subjects: “…Stargardt disease…”
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  10. 10
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    Effective splicing restoration of a deep-intronic ABCA4 variant in cone photoreceptor precursor cells by CRISPR/SpCas9 approaches by Pietro De Angeli, Peggy Reuter, Stefan Hauser, Ludger Schöls, Katarina Stingl, Bernd Wissinger, Susanne Kohl

    Published 2022
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  11. 11
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    Formulation and Evaluation of SNEDDS Loaded with Original Lipophenol for the Oral Route to Prevent Dry AMD and Stragardt's Disease by Maxime Vincent, Laurianne Simon, Philippe Brabet, Philippe Legrand, Christophe Dorandeu, Josephine Lai Kee Him, Thierry Durand, Céline Crauste, Sylvie Begu

    Published 2022
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  12. 12
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    Lentiviral Vectors for Ocular Gene Therapy by Yvan Arsenijevic, Adeline Berger, Florian Udry, Corinne Kostic

    Published 2022
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  13. 13
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    Morphological and physiological retinal degeneration induced by intravenous delivery of vitamin A dimers in rabbits by Jackie Penn, Doina M. Mihai, Ilyas Washington

    Published 2015
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  14. 14
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    Ophthalmology: Current and Future Developments: Volume 2 - Diagnostic Atlas of Retinal Diseases

    Published 2017
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  15. 15
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    Implementation of experimental cellular (cellular-genetic) therapies on the example of eye diseases by Patrycja Kapczuk, Karolina Rogulska, Anna Stangret, Agata Mularczyk, Angelika Szczęśniak, Katarzyna Topczewska, Konrad Grzeszczak

    Published 2020
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  16. 16
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    Ophthalmology: Current and Future Developments: Volume 2 - Diagnostic Atlas of Retinal Diseases

    Published 2017
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  17. 17
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    Lipopeptide-mediated Cas9 RNP delivery: A promising broad therapeutic strategy for safely removing deep-intronic variants in ABCA4 by Irene Vázquez-Domínguez, Mert Öktem, Florian A. Winkelaar, Thai Hoang Nguyen, Anita D.M. Hoogendoorn, Eleonora Roschi, Galuh D.N. Astuti, Raoul Timmermans, Nuria Suárez-Herrera, Ilaria Bruno, Albert Ruiz-Llombart, Joseph Brealey, Olivier G. de Jong, Rob W.J. Collin, Enrico Mastrobattista, Alejandro Garanto

    Published 2024
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  18. 18
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    The P2Y12 Receptor Antagonist Ticagrelor Reduces Lysosomal pH and Autofluorescence in Retinal Pigmented Epithelial Cells From the ABCA4-/- Mouse Model of Retinal Degeneration by Wennan Lu, Néstor M. Gómez, Jason C. Lim, Sonia Guha, Sonia Guha, Ann O'Brien-Jenkins, Erin E. Coffey, Keith E. Campagno, Stuart A. McCaughey, Alan M. Laties, Leif G. Carlsson, Claire H. Mitchell, Claire H. Mitchell, Claire H. Mitchell

    Published 2018
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