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RNAi-mediated silencing of hepatic Alas1 effectively prevents and treats the induced acute attacks in acute intermittent porphyria mice.
Yasuda, Makiko; Gan, Lin; Chen, Brenden; Kadirvel, Senkottuvelan; Yu, Chunli; Phillips, John D; New, Maria I; Liebow, Abigail; Fitzgerald, Kevin; Querbes, William; Desnick, Robert J.
Afiliação
  • Yasuda M; Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York, NY 10029;
  • Gan L; Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York, NY 10029;
  • Chen B; Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York, NY 10029;
  • Kadirvel S; Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York, NY 10029;
  • Yu C; Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York, NY 10029;
  • Phillips JD; Hematology Division, University of Utah School of Medicine, Salt Lake City, UT 84132;
  • New MI; Department of Pediatrics, Icahn School of Medicine at Mount Sinai, New York, NY 10029; and maria.new@mssm.edu robert.desnick@mssm.edu.
  • Liebow A; Alnylam Pharmaceuticals, Cambridge, MA 02142.
  • Fitzgerald K; Alnylam Pharmaceuticals, Cambridge, MA 02142.
  • Querbes W; Alnylam Pharmaceuticals, Cambridge, MA 02142.
  • Desnick RJ; Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York, NY 10029; maria.new@mssm.edu robert.desnick@mssm.edu.
Proc Natl Acad Sci U S A ; 111(21): 7777-82, 2014 May 27.
Article em En | MEDLINE | ID: mdl-24821812
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Porfiria Aguda Intermitente / RNA Interferente Pequeno / Interferência de RNA / 5-Aminolevulinato Sintetase / Fígado Tipo de estudo: Prognostic_studies Limite: Animals Idioma: En Revista: Proc Natl Acad Sci U S A Ano de publicação: 2014 Tipo de documento: Article
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Porfiria Aguda Intermitente / RNA Interferente Pequeno / Interferência de RNA / 5-Aminolevulinato Sintetase / Fígado Tipo de estudo: Prognostic_studies Limite: Animals Idioma: En Revista: Proc Natl Acad Sci U S A Ano de publicação: 2014 Tipo de documento: Article