Van Diepen, Hester Catharina,Chan, Hee Lam,Turunen, Janne Juha
申请号：
AU2017257292
公开号：
AU2017257292A1
申请日:
2017.04.25
申请国别(地区):
AU
年份:
2018
代理人:
摘要：
The invention relates to the fields of medicine and immunology. In particular, it relates to novel antisense oligonucleotides that may be used in the treatment, prevention and/or delay of Usher Syndrome type II and/or USH2A-associated non syndromic retina degeneration, especially by skipping a pseudo exon (PE40) between exon 40 and 41 in the human USH2Agene.
