发明名称 |
SMN2 ELEMENT 1 ANTISENSE COMPOSITIONS AND METHODS AND USES THEREOF |
摘要 |
The invention provides methods and compositions for treatment of spinal muscular atrophy (SMA). In one aspect of the invention, a series of compositions comprising an antisense oligonucleotide targeting the Element 1 site on the SMN2 pre-mRNA and a Morpholino backbone is disclosed. In another aspect of the invention, a method of treating SMA patients by modulating the splicing of SMN2 pre-mRNA to increase the amount of full-length SMN is disclosed. Certain embodiments of the inventive method comprise administering an E1-targeting antisense oligonucleotide, such as Morpholino based antisense oligonucleotide, to a SMA subject. |
申请公布号 |
WO2014169243(A2) |
申请公布日期 |
2014.10.16 |
申请号 |
WO2014US33856 |
申请日期 |
2014.04.11 |
申请人 |
THE CURATORS OF THE UNIVERSITY OF MISSOURI |
发明人 |
LORSON, CHRISTIAN;OSMAN, ERKAN |
分类号 |
A61K48/00;C07F9/6533 |
主分类号 |
A61K48/00 |
代理机构 |
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代理人 |
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主权项 |
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地址 |
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