BICISTRONIC AAV VECTOR FOR RNA INTERFERENCE IN ALS (Englisch)

Freier Zugriff

The present invention relates to a bicistronic expression vector for silencing a gene specifically in astrocytes and neurons, comprising two expression cassettes comprising a first and a second silencer sequence, respectively, wherein the expression of said first silencer sequence within astrocytes is regulated by an astrocyte-specific promoter and the expression of said second silencer sequence within neurons is regulated by a neuron-specific promoter. In a preferred embodiment, said first and second silencer sequences are SOD1 silencer sequences. Pharmaceutical composition comprising said bicistronic vector and the use of the same in the treatment of motoneuron diseases are further described.

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Dokumentinformationen

  • Titel:
    BICISTRONIC AAV VECTOR FOR RNA INTERFERENCE IN ALS
  • Patentnummer:
    US2018228919
  • Anmeldedatum:
    2016-07-22
  • Patentanmelder:
  • Patentfamilie:
  • Autor / Urheber:
  • Verlag:
    Europäisches Patentamt
  • Erscheinungsjahr:
    2015
  • Medientyp:
    Patent
  • Format:
    Elektronische Ressource
  • Sprache:
    Englisch
  • Klassifikation:
    IPC:    A61K Zubereitungen für medizinische, zahnärztliche oder kosmetische Zwecke, PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES / C12N Mikroorganismen oder Enzyme, MICROORGANISMS OR ENZYMES

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