Oligomers
First Claim
Patent Images
1. An oligomer for ameliorating DMD, the oligomer comprising at least 25 contiguous bases of base sequenceXGA AAA CGC CGC CAX XXC XCA ACA GAX CXG (SEQ ID NO:
- 1);
wherein X=U or T, wherein the oligomer'"'"'s base sequence can vary from the above sequence at up to two base positions, and wherein the molecule can bind to a target site to cause exon skipping in an exon of the dystrophin gene.
1 Assignment
0 Petitions
Accused Products
Abstract
Molecules are provided for inducing or facilitating exon skipping in forming spliced mRNA products from pre-mRNA molecules in cells. The molecules may be provided directly as oligonucleotides or expression products of vectors that are administered to a subject. High rates of skipping can be achieved. High rates of skipping reduce the severity of a disease like Duchene Muscular Dystrophy so that the disease is more like Becker Muscular Dystrophy. This is a severe reduction in symptom severity and mortality.
63 Citations
10 Claims
-
1. An oligomer for ameliorating DMD, the oligomer comprising at least 25 contiguous bases of base sequence
XGA AAA CGC CGC CAX XXC XCA ACA GAX CXG (SEQ ID NO: - 1);
wherein X=U or T, wherein the oligomer'"'"'s base sequence can vary from the above sequence at up to two base positions, and wherein the molecule can bind to a target site to cause exon skipping in an exon of the dystrophin gene. - View Dependent Claims (2, 3, 4, 5, 6, 7, 8, 9, 10)
- 1);
Specification