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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:
EP16306575.8
Publications:
EMBO Mol Med. 2018 Feb;10(2):239-253. doi: 10.15252/emmm.201707988.
Reference:
BIO17148-T1
Application:
Therapeutic
Therapy Area:
Genetic Disorders
Type of patent:
Product
Field of development:
Drug
Stage of development:
Hit - validation in vivo
Type of Product:
Rnai gene therapy
Business Developper
contact
Grégoire SERRA
Business Developer
Phone
E-mail
Patent filling date:
2016-11-29
Rare disease:
Yes
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