Title:
MODIFIED ANTISENSE OLIGOMERS FOR EXON INCLUSION IN SPINAL MUSCULAR ATROPHY
Document Type and Number:
Japanese Patent JP2021048877
Kind Code:
A
Abstract:
To provide modified antisense oligomers for exon inclusion in spinal muscular atrophy.SOLUTION: The present disclosure relates to modified antisense oligomers and related compositions and methods for increasing the expression of functional SMN protein and for treating spinal muscular atrophy, and relates also to inducing inclusion of exon 7 in SMN2 mRNA. In certain aspects, the present disclosure provides compositions and methods for increasing the expression of functional SMN protein. In further aspects, the present disclosure provides variously described modified antisense oligomers for enhancing levels of exon 7-containing SMN2 mRNA in a subject.SELECTED DRAWING: None
Inventors:
MARCO A PASSINI
GUNNAR J HANSON
GUNNAR J HANSON
Application Number:
JP2020216610A
Publication Date:
April 01, 2021
Filing Date:
December 25, 2020
Export Citation:
Assignee:
SAREPTA THERAPEUTICS INC
International Classes:
C12N15/113; A61K31/7088; A61K48/00; A61P21/00; A61P25/00
Attorney, Agent or Firm:
Takeshi Oshio