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Gene therapy for tuberous sclerosis complex type 2 in a mouse model by delivery of AAV9 encoding a condensed form of tuberin.
Cheah, Pike-See; Prabhakar, Shilpa; Yellen, David; Beauchamp, Roberta L; Zhang, Xuan; Kasamatsu, Shingo; Bronson, Roderick T; Thiele, Elizabeth A; Kwiatkowski, David J; Stemmer-Rachamimov, Anat; György, Bence; Ling, King-Hwa; Kaneki, Masao; Tannous, Bakhos A; Ramesh, Vijaya; Maguire, Casey A; Breakefield, Xandra O.
Afiliación
  • Cheah PS; Molecular Neurogenetics Unit, Department of Neurology and Center for Molecular Imaging Research, Department of Radiology, Massachusetts General Hospital, and Program in Neuroscience, Harvard Medical School, Boston, MA, USA.
  • Prabhakar S; Department of Human Anatomy, Faculty of Medicine and Health Sciences, Universiti Putra Malaysia, Serdang, Malaysia.
  • Yellen D; Molecular Neurogenetics Unit, Department of Neurology and Center for Molecular Imaging Research, Department of Radiology, Massachusetts General Hospital, and Program in Neuroscience, Harvard Medical School, Boston, MA, USA.
  • Beauchamp RL; Molecular Neurogenetics Unit, Department of Neurology and Center for Molecular Imaging Research, Department of Radiology, Massachusetts General Hospital, and Program in Neuroscience, Harvard Medical School, Boston, MA, USA.
  • Zhang X; Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.
  • Kasamatsu S; Molecular Neurogenetics Unit, Department of Neurology and Center for Molecular Imaging Research, Department of Radiology, Massachusetts General Hospital, and Program in Neuroscience, Harvard Medical School, Boston, MA, USA.
  • Bronson RT; Department of Anesthesia, Critical Care and Pain Medicine, Massachusetts General Hospital, Harvard Medical School, Charlestown, MA, USA.
  • Thiele EA; Shriners Hospitals for Children, Boston, MA, USA.
  • Kwiatkowski DJ; Rodent Histopathology Core Facility, Harvard Medical School, Boston, MA, USA.
  • Stemmer-Rachamimov A; Herscot Center for Tuberous Sclerosis Complex, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.
  • György B; The Pediatric Epilepsy Program, Department of Neurology, Massachusetts General Hospital, Boston, MA, USA.
  • Ling KH; Brigham and Women's Hospital, Harvard Medical School, Boston, MA, USA.
  • Kaneki M; Department of Pathology, Massachusetts General Hospital, Boston, MA, USA.
  • Tannous BA; Department of Neurobiology and Howard Hughes Medical Institute, Harvard Medical School, Boston, MA, USA.
  • Ramesh V; Institute of Molecular and Clinical Ophthalmology, Basel, Switzerland.
  • Maguire CA; Department of Ophthalmology, University of Basel, Basel, Switzerland.
  • Breakefield XO; Department of Genetics, Harvard Medical School, Boston, MA, USA.
Sci Adv ; 7(2)2021 01.
Article en En | MEDLINE | ID: mdl-33523984
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Idioma: En Revista: Sci Adv Año: 2021 Tipo del documento: Article País de afiliación: Estados Unidos Pais de publicación: Estados Unidos
Texto completo
Añadir a Mi BVS
Imprimir
XML
PubMed Links
Buscar en Google

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Idioma: En Revista: Sci Adv Año: 2021 Tipo del documento: Article País de afiliación: Estados Unidos Pais de publicación: Estados Unidos