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Title:
脊髄性筋萎縮症における誘発されたエクソン包含
Document Type and Number:
Japanese Patent JP6496549
Kind Code:
B2
Abstract:
The invention relates to the use of an antisense compound for inducing exon inclusion as a treatment for Spinal Muscle Atrophy (SMA). More particularly it relates to inducing inclusion of exon 7 to restore levels of Survival Motor Neuron (SMN) protein encoded by the Survival Motor Neuron (SMN) gene.

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Inventors:
Linsley, Peter
Repertoire, Brian James
Application Number:
JP2014544962A
Publication Date:
April 03, 2019
Filing Date:
November 30, 2012
Export Citation:
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Assignee:
Saleputa Therapeutics, Incorporated
International Classes:
C12N15/113; A61K31/712; A61K31/7125; A61K48/00; A61P21/02
Domestic Patent References:
JP2010505741A
JP2003516965A
JP2011236157A
Foreign References:
WO2007002390A1
Other References:
RNA Biology,2009年,Vol. 6, No. 3,p. 341-350
Polymer,2003年,Vol. 44,p. 7299-7309
J. Am. Chem. Soc.,1994年,Vol. 116,p. 10914-10920
Attorney, Agent or Firm:
Hidesaku Yamamoto
Natsuki Morishita



 
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