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Research Article

Lentiviral Gene Transfer of Rpe65 Rescues Survival and Function of Cones in a Mouse Model of Leber Congenital Amaurosis

  • Alexis-Pierre Bemelmans equal contributor,

    equal contributor Contributed equally to this work with: Alexis-Pierre Bemelmans, Corinne Kostic

    Affiliation: Unit of Gene Therapy and Stem Cell Biology, Jules Gonin Eye Hospital, Lausanne, Switzerland

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  • Corinne Kostic equal contributor,

    equal contributor Contributed equally to this work with: Alexis-Pierre Bemelmans, Corinne Kostic

    Affiliation: Unit of Gene Therapy and Stem Cell Biology, Jules Gonin Eye Hospital, Lausanne, Switzerland

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  • Sylvain V Crippa,

    Affiliation: Unit of Gene Therapy and Stem Cell Biology, Jules Gonin Eye Hospital, Lausanne, Switzerland

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  • William W Hauswirth,

    Affiliation: Department of Ophthalmology, University of Florida, Gainesville, Florida, United States of America

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  • Janis Lem,

    Affiliation: Department of Ophthalmology, Program in Genetics and Tufts Center for Vision Research, Tufts University School of Medicine, Boston, Massachusetts, United States of America

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  • Francis L Munier,

    Affiliation: Unit of Clinical Oculogenetics, Jules Gonin Eye Hospital, Lausanne, Switzerland

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  • Mathias W Seeliger,

    Affiliation: Retinal Electrodiagnostics Research Group, Department of Ophthalmology II, Eberhard-Karls University, Tübingen, Germany

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  • Andreas Wenzel,

    Affiliation: Laboratory of Retinal Cell Biology, University Hospital, Zürich, Switzerland

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  • Yvan Arsenijevic mail

    To whom correspondence should be addressed. E-mail: yvan.arsenijevic@ophtal.vd.ch

    Affiliation: Unit of Gene Therapy and Stem Cell Biology, Jules Gonin Eye Hospital, Lausanne, Switzerland

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  • Published: October 10, 2006
  • DOI: 10.1371/journal.pmed.0030347

About the Authors

Alexis-Pierre Bemelmans, Corinne Kostic, Sylvain V Crippa, Yvan Arsenijevic
Unit of Gene Therapy and Stem Cell Biology, Jules Gonin Eye Hospital, Lausanne, Switzerland
William W Hauswirth
Department of Ophthalmology, University of Florida, Gainesville, Florida, United States of America
Janis Lem
Department of Ophthalmology, Program in Genetics and Tufts Center for Vision Research, Tufts University School of Medicine, Boston, Massachusetts, United States of America
Francis L Munier
Unit of Clinical Oculogenetics, Jules Gonin Eye Hospital, Lausanne, Switzerland
Mathias W Seeliger
Retinal Electrodiagnostics Research Group, Department of Ophthalmology II, Eberhard-Karls University, Tübingen, Germany
Andreas Wenzel
Laboratory of Retinal Cell Biology, University Hospital, Zürich, Switzerland

Corresponding Author

Email: yvan.arsenijevic@ophtal.vd.ch

Competing Interests

The authors have declared that no competing interests exist.

Author Contributions

APB, CK, AW, and YA designed the study. APB, CK, SVC, FLM, MWS, AW, and YA analyzed the data. APB, CK, FLM, AW, and YA contributed to writing the paper. WWH provided the R0.8 promoter used. JL created the Gnat1−/− transgenic mouse models used in the study.