Antisense oligonucleotides for the treatment of stargardt disease

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专利权人:

ProQR Therapeutics II B.V.

发明人:

Yilmaz-Elis, Aliye Seda,Adamson, Peter,Dulla, Kalyana Chakravarthi,Schulkens, Iris Antoinette Ernestine

申请号：

AU2018252191

公开号：

AU2018252191A1

申请日:

2018.04.13

申请国别(地区):

AU

年份:

2019

代理人:

摘要：

The invention relates to the fields of medicine and biotechnology. In particular, it relates to novel antisense oligonucleotides (AONs) that may be used in the treatment, prevention and/or delay of Stargardt disease and/or ABCA4-associated eye disease. More in particular, the invention relates to AONs that are used in inhibiting or blocking exon 39 skipping in the human ABCA4 pre-mRNA.

来源网站：

中国工程科技知识中心

来源网址：

http://www.ckcest.cn/home/