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Open Access
Peer-reviewed
Research Article
AAV-Mediated Gene Therapy for Choroideremia: Preclinical Studies in Personalized Models
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Vidyullatha Vasireddy,
Affiliation F.M. Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania School of Medicine, Philadelphia, Pennsylvania, United States of America
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Jason A. Mills ,
Contributed equally to this work with: Jason A. Mills, Rajashekhar Gaddameedi, Etiena Basner-Tschakarjan
Affiliation Center for Cellular and Molecular Therapeutics, The Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, United States of America
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Rajashekhar Gaddameedi ,
Contributed equally to this work with: Jason A. Mills, Rajashekhar Gaddameedi, Etiena Basner-Tschakarjan
Affiliation F.M. Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania School of Medicine, Philadelphia, Pennsylvania, United States of America
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Etiena Basner-Tschakarjan ,
Contributed equally to this work with: Jason A. Mills, Rajashekhar Gaddameedi, Etiena Basner-Tschakarjan
Affiliation Center for Cellular and Molecular Therapeutics, The Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, United States of America
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Monika Kohnke,
Affiliation Institute for Molecular Bioscience, University of Queensland, St. Lucia, Queensland, Australia
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Aaron D. Black,
Affiliation F.M. Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania School of Medicine, Philadelphia, Pennsylvania, United States of America
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Krill Alexandrov,
Affiliation Institute for Molecular Bioscience, University of Queensland, St. Lucia, Queensland, Australia
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Shangzhen Zhou,
Affiliation Center for Cellular and Molecular Therapeutics, The Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, United States of America
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Albert M. Maguire,
Affiliations F.M. Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania School of Medicine, Philadelphia, Pennsylvania, United States of America, Center for Cellular and Molecular Therapeutics, The Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, United States of America
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Daniel C. Chung,
Affiliations F.M. Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania School of Medicine, Philadelphia, Pennsylvania, United States of America, Center for Cellular and Molecular Therapeutics, The Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, United States of America
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Helen Mac,
Affiliation Center for Cellular and Molecular Therapeutics, The Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, United States of America
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Lisa Sullivan,
Affiliation Department of Anatomic Pathology, The Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, United States of America
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Paul Gadue,
Affiliations Center for Cellular and Molecular Therapeutics, The Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, United States of America, Department of Pathology and Laboratory Medicine, The Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, United States of America
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Jeannette L. Bennicelli,
Affiliation F.M. Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania School of Medicine, Philadelphia, Pennsylvania, United States of America
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Deborah L. French,
Affiliations Center for Cellular and Molecular Therapeutics, The Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, United States of America, Department of Pathology and Laboratory Medicine, The Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, United States of America
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Jean Bennett
* E-mail: jebennet@mail.med.upenn.edu
Affiliations F.M. Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania School of Medicine, Philadelphia, Pennsylvania, United States of America, Center for Cellular and Molecular Therapeutics, The Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, United States of America
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AAV-Mediated Gene Therapy for Choroideremia: Preclinical Studies in Personalized Models
- Vidyullatha Vasireddy,
- Jason A. Mills,
- Rajashekhar Gaddameedi,
- Etiena Basner-Tschakarjan,
- Monika Kohnke,
- Aaron D. Black,
- Krill Alexandrov,
- Shangzhen Zhou,
- Albert M. Maguire,
- Daniel C. Chung
- Published: May 7, 2013
- https://doi.org/10.1371/journal.pone.0061396