pre-mrna splicing switching and modulation comprising bicyclic framework portions, with improved characteristics for the treatment of genetic disorders
JUDITH CHRISTINA THEODORA VAN DEUTEKOM,PETER CHRISTIAN DE VISSER
申请号：
BR112019000061
公开号：
BR112019000061A2
申请日:
2017.07.05
申请国别(地区):
BR
年份:
2019
代理人:
摘要：
The present invention provides antisense splicing switching oligonucleotides with improved characteristics that enhance clinical applicability for treatment, amelioration, prevention and / or delay of neuromuscular disorders, such as dmd.a presente invenção fornece oligonucleotídeos de comutação de splicing antissentido com características melhoradas que intensificam a aplicabilidade clínica para tratamento, melhoria, prevenção e/ou atraso de distúrbios neuromusculares, tal como dmd.
