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Open Access
Peer-reviewed
Research Article
Pharmacological and rAAV Gene Therapy Rescue of Visual Functions in a Blind Mouse Model of Leber Congenital Amaurosis
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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
*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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Pharmacological and rAAV Gene Therapy Rescue of Visual Functions in a Blind Mouse Model of Leber Congenital Amaurosis
- Matthew L Batten,
- Yoshikazu Imanishi,
- Daniel C Tu,
- Thuy Doan,
- Li Zhu,
- Jijing Pang,
- Lyudmila Glushakova,
- Alexander R Moise,
- Wolfgang Baehr,
- Russell N. Van Gelder
- Published: November 1, 2005
- https://doi.org/10.1371/journal.pmed.0020333