发明名称 |
Means for efficient skipping of exon 45 in dystrophin pre-mrna using an antisense oligonucleotide and uses thereof |
摘要 |
<p>The. invention relates to a method for inducing or promoting skipping of exon 45 of DMD pre-mRNA in a Duchenne Muscular Dystrophy patient, preferably in an isolated (muscle) cell, the method comprising providing said cell with an antisense molecule that binds to a continuous stretch of at least 21 nucleotides within said exon. The invention further relates to such antisense molecule used in said method.</p> |
申请公布号 |
IL212508(A) |
申请公布日期 |
2013.11.28 |
申请号 |
IL20110212508 |
申请日期 |
2011.04.27 |
申请人 |
ACADEMISCH ZIEKENHUIS LEIDEN;PROSENSA TECHNOLOGIES B.V.;JUDITH CHRISTINA THEODORA VAN DEUTEKOM;ANNEMIEKE AARTSMA-RUS;JOSEPHUS JOHANNES DE KIMPE;GARRIT-JAN BOUDEWIJN VAN OMMEN;PROSENSA B.V.;PROSENSA HOLDING B.V.;GERADUS JOHANNES PLATENBURG |
发明人 |
JUDITH CHRISTINA THEODORA VAN DEUTEKOM;ANNEMIEKE AARTSMA-RUS;JOSEPHUS JOHANNES DE KIMPE;GARRIT-JAN BOUDEWIJN VAN OMMEN;GERADUS JOHANNES PLATENBURG |
分类号 |
A61K;C12N |
主分类号 |
A61K |
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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