Hester Catharina Van Diepen,Hee Lam Chan,Janne Juha Turunen
申请号:
US16811848
公开号:
US20200237802A1
申请日:
2020.03.06
申请国别(地区):
US
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 USH2Agene.