The patent badge is an abbreviated version of the USPTO patent document. The patent badge does contain a link to the full patent document.

The patent badge is an abbreviated version of the USPTO patent document. The patent badge covers the following: Patent number, Date patent was issued, Date patent was filed, Title of the patent, Applicant, Inventor, Assignee, Attorney firm, Primary examiner, Assistant examiner, CPCs, and Abstract. The patent badge does contain a link to the full patent document (in Adobe Acrobat format, aka pdf). To download or print any patent click here.

Date of Patent:
Nov. 17, 2020

Filed:

Jul. 22, 2016
Applicant:

Ecole Polytechnique Federale DE Lausanne (Epfl), Lausanne, CH;

Inventors:

Julianne Aebischer, Lausanne, CH;

Bernard Schneider, Lausanne, CH;

Cylia Rochat, Lausanne, CH;

Attorney:
Primary Examiner:
Int. Cl.
CPC ...
A61K 48/00 (2006.01); C12N 15/86 (2006.01); C12N 15/113 (2010.01); C12N 15/79 (2006.01);
U.S. Cl.
CPC ...
A61K 48/0058 (2013.01); C12N 15/113 (2013.01); C12N 15/79 (2013.01); C12N 15/86 (2013.01); C12N 2750/14141 (2013.01); C12N 2750/14143 (2013.01); C12N 2830/008 (2013.01); C12N 2830/20 (2013.01); C12N 2830/42 (2013.01); C12N 2830/48 (2013.01); C12N 2830/50 (2013.01);
Abstract

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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