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Artificial MicroRNAs Targeting C9orf72 Can Reduce Accumulation of Intra-nuclear Transcripts in ALS and FTD Patients.
Martier, Raygene; Liefhebber, Jolanda M; Miniarikova, Jana; van der Zon, Tom; Snapper, Jolanda; Kolder, Iris; Petry, Harald; van Deventer, Sander J; Evers, Melvin M; Konstantinova, Pavlina.
Afiliación
  • Martier R; Department of Research & Development, uniQure Biopharma B.V., Amsterdam, the Netherlands; Department of Gastroenterology and Hepatology, Leiden University Medical Center, Leiden, the Netherlands.
  • Liefhebber JM; Department of Research & Development, uniQure Biopharma B.V., Amsterdam, the Netherlands.
  • Miniarikova J; Department of Research & Development, uniQure Biopharma B.V., Amsterdam, the Netherlands.
  • van der Zon T; Department of Research & Development, uniQure Biopharma B.V., Amsterdam, the Netherlands.
  • Snapper J; Department of Research & Development, uniQure Biopharma B.V., Amsterdam, the Netherlands.
  • Kolder I; BaseClear B.V., Sylviusweg 74, 2333 BE, Leiden, the Netherlands.
  • Petry H; Department of Research & Development, uniQure Biopharma B.V., Amsterdam, the Netherlands.
  • van Deventer SJ; Department of Research & Development, uniQure Biopharma B.V., Amsterdam, the Netherlands; Department of Gastroenterology and Hepatology, Leiden University Medical Center, Leiden, the Netherlands.
  • Evers MM; Department of Research & Development, uniQure Biopharma B.V., Amsterdam, the Netherlands.
  • Konstantinova P; Department of Research & Development, uniQure Biopharma B.V., Amsterdam, the Netherlands. Electronic address: p.konstantinova@uniqure.com.
Mol Ther Nucleic Acids ; 14: 593-608, 2019 Mar 01.
Article en En | MEDLINE | ID: mdl-30776581
Palabras clave

Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Idioma: En Revista: Mol Ther Nucleic Acids Año: 2019 Tipo del documento: Article País de afiliación: Países Bajos

Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Idioma: En Revista: Mol Ther Nucleic Acids Año: 2019 Tipo del documento: Article País de afiliación: Países Bajos