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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 Marc,PRUDHON Bernard,TROCHET Delphine

Publications:

EMBO Mol Med. 2018 Feb;10(2):239-253. doi: 10.15252/emmm.201707988.

Reference:

BIO17148-T1

Inserm Transfert
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Patent filling date: 2016-11-29

Rare disease: Yes

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