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Rescue of a familial dysautonomia mouse model by AAV9-Exon-specific U1 snRNA.
Romano, Giulia; Riccardi, Federico; Bussani, Erica; Vodret, Simone; Licastro, Danilo; Ragone, Isabella; Ronzitti, Giuseppe; Morini, Elisabetta; Slaugenhaupt, Susan A; Pagani, Franco.
Afiliação
  • Romano G; Human Molecular Genetics Laboratory, International Centre for Genetic Engineering and Biotechnology, Padriciano, 34149 Trieste, Italy.
  • Riccardi F; Human Molecular Genetics Laboratory, International Centre for Genetic Engineering and Biotechnology, Padriciano, 34149 Trieste, Italy.
  • Bussani E; Human Molecular Genetics Laboratory, International Centre for Genetic Engineering and Biotechnology, Padriciano, 34149 Trieste, Italy.
  • Vodret S; Human Molecular Genetics Laboratory, International Centre for Genetic Engineering and Biotechnology, Padriciano, 34149 Trieste, Italy.
  • Licastro D; Area Science Park, Padriciano, 34149 Trieste, Italy.
  • Ragone I; Genethon, 91000 Evry, France; Université Paris-Saclay, Univ Evry, Inserm, Genethon, Integrare research Unit UMR_S951, 91000 Evry, France.
  • Ronzitti G; Genethon, 91000 Evry, France; Université Paris-Saclay, Univ Evry, Inserm, Genethon, Integrare research Unit UMR_S951, 91000 Evry, France.
  • Morini E; Center for Genomic Medicine, Massachusetts General Hospital Research Institute, Boston, MA, USA; Department of Neurology, Massachusetts General Hospital Research Institute and Harvard Medical School, Boston, MA, USA.
  • Slaugenhaupt SA; Center for Genomic Medicine, Massachusetts General Hospital Research Institute, Boston, MA, USA; Department of Neurology, Massachusetts General Hospital Research Institute and Harvard Medical School, Boston, MA, USA.
  • Pagani F; Human Molecular Genetics Laboratory, International Centre for Genetic Engineering and Biotechnology, Padriciano, 34149 Trieste, Italy; Dipartimento di Scienze della Vita e Biotecnologie, Università di Ferrara, Ferrara, Italy. Electronic address: franco.pagani@icgeb.org.
Am J Hum Genet ; 109(8): 1534-1548, 2022 08 04.
Article em En | MEDLINE | ID: mdl-35905737
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Disautonomia Familiar / Doenças Neurodegenerativas Limite: Animals / Humans Idioma: En Ano de publicação: 2022 Tipo de documento: Article
Texto completo
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XML
PubMed Links
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Disautonomia Familiar / Doenças Neurodegenerativas Limite: Animals / Humans Idioma: En Ano de publicação: 2022 Tipo de documento: Article