公开(公告)号：US11872290B2

公开(公告)日：2024-01-16

申请号：US17080205

申请日：2020-10-26

申请人： Ecole Polytechnique Federale De Lausanne (EPFL)

发明人： Julianne Aebischer ,  Bernard Schneider ,  Cylia Rochat

IPC分类号： A61K48/00 ,  C12N15/86 ,  C12N15/113 ,  C12N15/79

CPC分类号： A61K48/0058 ,  C12N15/113 ,  C12N15/79 ,  C12N15/86 ,  C12N2750/14141 ,  C12N2750/14143 ,  C12N2830/008 ,  C12N2830/20 ,  C12N2830/42 ,  C12N2830/48 ,  C12N2830/50

摘要： 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.

公开(公告)号：US20210052744A1

公开(公告)日：2021-02-25

申请号：US17080205

申请日：2020-10-26

申请人： Ecole Polytechnique Federale De Lausanne (EPFL)

发明人： Julianne Aebischer ,  Bernard Schneider ,  Cylia Rochat

IPC分类号： A61K48/00 ,  C12N15/113 ,  C12N15/79 ,  C12N15/86

摘要： 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.

公开(公告)号：US20180228919A1

公开(公告)日：2018-08-16

申请号：US15746458

申请日：2016-07-22

申请人： Ecole Polytechnique Federale De Lausanne (EPFL)

发明人： Julianne Aebischer ,  Bernard Schneider ,  Cylia Rochat

IPC分类号： A61K48/00 ,  C12N15/79 ,  C12N15/113

CPC分类号： A61K48/0058 ,  C12N15/113 ,  C12N15/79 ,  C12N15/86 ,  C12N2750/14141 ,  C12N2750/14143 ,  C12N2830/008 ,  C12N2830/20 ,  C12N2830/42 ,  C12N2830/48 ,  C12N2830/50

摘要： 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.