VAN DIEPEN, Hester Catharina,CHAN, Hee Lam,TURUNEN, Janne Juha
申请号：
DK17721984
公开号：
DK3448999T3
申请日:
2017.04.25
申请国别(地区):
DK
年份:
2020
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 USH2A gene.
