MUTANTS OF SRSF1 (ASF/SF2) FOR TREATING SPINAL MUSCULAR ATROPHY

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专利权人:

发明人:

申请号：

EP15165741.8

公开号：

EP3087995A1

申请日:

2015.04.29

申请国别(地区):

EP

年份:

2016

代理人:

摘要：

The invention relates to non-naturally occurring proteins with a splicing factor activity that are able to facilitate the inclusion of exon 7 of the SMN1 and/or SMN2 gene into the mature mRNA. The disclosed non-naturally occurring proteins share at least 70% sequence identity with hSRSF1 (ASF/SF2) and can be used in the treatment of spinal muscular atrophy.

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