METHODS AND COMPOSITIONS FOR DYSFERLIN EXON-SKIPPING
摘要
The disclosure provides methods and compositions for inducing exon-skipping in a dysferlin pre-mRNA useful, e.g., in restoring function in a dysferlin deficiency. The disclosure also provides improved methods and compositions for generally inducing exon-skipping in a pre-mRNA.
申请公布号
EP2494049(A2)
申请公布日期
2012.09.05
申请号
EP20100781537
申请日期
2010.10.29
申请人
ACADEMISCH ZIEKENHUIS LEIDEN H.O.D.N. LUMC
发明人
VAN DER MAAREL, SILVERE MARIA;VAN OMMEN, GARRIT-JAN, BOUDEWIJN;AARTSMA-RUS, ANNEMIEKE;HOUWELING-GAZZOLI, ISABELLA;DEN DUNNEN, JOHANNES, THEODORUS