ANTISENSE COMPOUNDS AND USES THEREOF

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专利权人:

Ionis Pharmaceuticals; Inc.

发明人:

RIGO, Frank,HASTINGS, Michelle, L.

申请号：

EP14867872

公开号：

EP3077510A4

申请日:

2014.12.02

申请国别(地区):

EP

年份:

2017

代理人:

摘要：

The present invention provides compounds comprising oligonucleotides complementary to a CLN3 transcript. Certain such compounds are useful for hybridizing to a CLN3 transcript, including but not limited to a CLN3 transcript in a cell. In certain embodiments, such hybridization results in modulation of splicing of the CLN3 transcript. In certain embodiments, such compounds are used to treat one or more symptoms associated with Batten Disease.

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