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Research Article

Pharmacological and rAAV Gene Therapy Rescue of Visual Functions in a Blind Mouse Model of Leber Congenital Amaurosis

  • Matthew L Batten,

    Affiliation: Department of Ophthalmology, University of Washington, Seattle, Washington, United States of America

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  • Yoshikazu Imanishi,

    Affiliations: Department of Ophthalmology, University of Washington, Seattle, Washington, United States of America, Department of Pharmacology, Case School of Medicine, Case Western Reserve University, Cleveland, Ohio, United States of America

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  • Daniel C Tu,

    Affiliation: Department of Ophthalmology and Visual Sciences, Washington University School of Medicine, St. Louis, Missouri, United States of America

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  • Thuy Doan,

    Affiliation: Department of Physiology and Biophysics, University of Washington, Seattle, Washington, United States of America

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  • Li Zhu,

    Affiliations: Department of Ophthalmology, University of Washington, Seattle, Washington, United States of America, Department of Chemistry, University of Washington, Seattle, Washington, United States of America

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  • Jijing Pang,

    Affiliation: Department of Ophthalmology, and Powell Gene Therapy Center, University of Florida, Gainesville, Florida, United States of America

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  • Lyudmila Glushakova,

    Affiliation: Department of Ophthalmology, and Powell Gene Therapy Center, University of Florida, Gainesville, Florida, United States of America

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  • Alexander R Moise,

    Affiliations: Department of Ophthalmology, University of Washington, Seattle, Washington, United States of America, Department of Pharmacology, Case School of Medicine, Case Western Reserve University, Cleveland, Ohio, United States of America

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  • Wolfgang Baehr,

    Affiliations: Department of Ophthalmology, University of Utah, Salt Lake City, Utah, United States of America, Department of Biology, University of Utah, Salt Lake City, Utah, United States of America, Department of Neurobiology and Anatomy, University of Utah, Salt Lake City, Utah, United States of America

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  • Russell N. Van Gelder,

    Affiliations: Department of Ophthalmology and Visual Sciences, Washington University School of Medicine, St. Louis, Missouri, United States of America, Department of Molecular Biology, Washington University School of Medicine, St. Louis, Missouri, United States of America, Department of Pharmacology, Washington University School of Medicine, St. Louis, Missouri, United States of America

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  • William W Hauswirth,

    Affiliation: Department of Ophthalmology, and Powell Gene Therapy Center, University of Florida, Gainesville, Florida, United States of America

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  • Fred Rieke,

    Affiliation: Department of Physiology and Biophysics, University of Washington, Seattle, Washington, United States of America

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  • Krzysztof Palczewski mail

    To whom correspondence should be addressed. E-mail: kxp65@case.edu

    Affiliations: Department of Ophthalmology, University of Washington, Seattle, Washington, United States of America, Department of Pharmacology, Case School of Medicine, Case Western Reserve University, Cleveland, Ohio, United States of America, Department of Chemistry, University of Washington, Seattle, Washington, United States of America, Department of Pharmacology, University of Washington, Seattle, Washington, United States of America

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  • Published: November 01, 2005
  • DOI: 10.1371/journal.pmed.0020333

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