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.

Keywords: Centronuclear Myopathies -
Patent Application number: EP16306575.8
Publications:
EMBO Mol Med. 2018 Feb;10(2):239-253. doi: 10.15252/emmm.201707988.

Reference:

BIO17148-T1

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Grégoire SERRA
Grégoire SERRA
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Patent filling date: 2016-11-29
Rare disease: Yes

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