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.
Type:
Grant
Filed:
February 3, 2021
Date of Patent:
March 12, 2024
Assignees:
ASSOICATION INSTITUT DE MYOLOGIE, INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE), SORBONNE UNIVERSITE, CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE
Inventors:
Marc Bitoun, Delphine Trochet, Bernard Prudhon