AAV-Mediated Gene Delivery in Adult GM1-Gangliosidosis Mice Corrects Lysosomal Storage in CNS and Improves Survival

• Rena C. Baek,

  Affiliation Biology Department, Boston College, Chestnut Hill, Massachusetts, United States of America

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• Marike L. D. Broekman,

  Affiliations Program in Neuroscience, Department of Neurology, Massachusetts General Hospital and Harvard Medical School, Charlestown, Massachusetts, United States of America, Department of Pharmacology and Anatomy, Rudolf Magnus Institute of Neuroscience, University Medical Center Utrecht, Utrecht, The Netherlands

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• Stanley G. Leroy,

  Affiliation Program in Neuroscience, Department of Neurology, Massachusetts General Hospital and Harvard Medical School, Charlestown, Massachusetts, United States of America

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• Laryssa A. Tierney,

  Affiliation Program in Neuroscience, Department of Neurology, Massachusetts General Hospital and Harvard Medical School, Charlestown, Massachusetts, United States of America

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• Michael A. Sandberg,

  Affiliation Berman-Gund Laboratory for the Study of Retinal Degenerations, Harvard Medical School, Massachusetts Eye and Ear Infirmary, Boston, Massachusetts, United States of America

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• Alessandra d'Azzo,

  Affiliation Department of Genetics, St. Jude Children's Research Hospital, Memphis, Tennessee, United States of America

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• Thomas N. Seyfried,

  Affiliation Biology Department, Boston College, Chestnut Hill, Massachusetts, United States of America

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• Miguel Sena-Esteves

  * E-mail: Miguel.Esteves@umassmed.edu

  Affiliation Program in Neuroscience, Department of Neurology, Massachusetts General Hospital and Harvard Medical School, Charlestown, Massachusetts, United States of America

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