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Allele-specific silencing therapy for dynamin 2-related diseases

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.
More information (PDF)
Keywords: Centronuclear Myopathies -
Patent Application number: European Procedure (Patents) (EPA) - 29 Nov. 2016 - 16306575.8
Inventors:
BITOUN MarcPRUDHON BernardTROCHET Delphine
Publications:
EMBO Mol Med. 2018 Feb;10(2):239-253. doi: 10.15252/emmm.201707988.

Reference:

BIO17148-T1

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    Inserm Transfert
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    Patent filling date: 29-11-2016
    Rare disease: Yes
    Second indication: No

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