Allele-specific silencing therapy for Dynamin 2-related diseases
AssigneesAssociation Institut De Myologie, Inserm (Institut National De La Sante Et De La Recherche Medicale), Sorbonne Universite, Centre National De La Recherche Scientifique
InventorsMarc Bitoun, Delphine Trochet, Bernard Prudhon
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Abstract
The invention relates to an allele specific siRNA able to silence the expression of only one allele of a heterozygous DNM2 gene, for treating diseases caused by heterozygous mutation and/or overexpression of Dynamin 2.
Claims13
An allele specific siRNA (AS-siRNA) that comprises a sequence mismatch and is able to silence the expression of only one allele of a heterozygous DNM2 gene in a cell, wherein the DNM2 gene is heterozygous for the presence of a non-pathological polymorphism, wherein the non-pathological polymorphism is rs2229920 (C or T) or rs12461992 (A or T), and wherein the AS-siRNA targets a region of the allele comprising said non-pathological polymorphism.
An AS-siRNA that comprises a sense strand selected from the group consisting of :
- SEQ ID NO:5, SEQ ID NO:6, SEQ ID NO:7, SEQ ID NO:8, SEQ ID NO:9 and SEQ ID NO:10.
Dependencies are inferred from the claim text.