Bicistronic aav vector for rna interference in als

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.


Year:
2017
Keywords:
Note:
Alternative title(s) : (de) Bicistronischer aav-vektor zur rna-interferenz in als (fr) Vecteur aav bicistronique pour l'interférence arn dans als
Other identifiers:
EPO Family ID: 53776350
Laboratories:




 Record created 2017-05-02, last modified 2020-10-25


Rate this document:

Rate this document:
1
2
3
 
(Not yet reviewed)