Modulation of exon recognition in pre-mRNA by interfering with the secondary RNA structure
First Claim
Patent Images
1. A method for generating an oligonucleotide or an equivalent thereof comprising:
- determining from a secondary structure of RNA from an exon, a region that assumes a structure that is hybridized to another part of said RNA (closed structure) and a region that is not hybridized in said structure (open structure); and
generating said oligonucleotide or said equivalent thereof, of which at least part of said oligonucleotide or said equivalent thereof is complementary to said closed structure and of which at least another part of said oligonucleotide or said equivalent thereof is complementary to said open structure.
1 Assignment
0 Petitions
Accused Products
Abstract
The invention provides a method for generating an oligonucleotide with which an exon may be skipped in a pre-mRNA and thus excluded from a produced mRNA thereof. Further provided are methods for altering the secondary structure of an mRNA to interfere with splicing processes and uses of the oligonucleotides and methods in the treatment of disease. Further provided are pharmaceutical compositions and methods and means for inducing skipping of several exons in a pre-mRNA.
-
Citations
51 Claims
-
1. A method for generating an oligonucleotide or an equivalent thereof comprising:
-
determining from a secondary structure of RNA from an exon, a region that assumes a structure that is hybridized to another part of said RNA (closed structure) and a region that is not hybridized in said structure (open structure); and
generating said oligonucleotide or said equivalent thereof, of which at least part of said oligonucleotide or said equivalent thereof is complementary to said closed structure and of which at least another part of said oligonucleotide or said equivalent thereof is complementary to said open structure. - View Dependent Claims (2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 22, 23, 24, 25, 26, 27, 28, 29, 30, 31, 32, 33, 34, 35, 36)
-
- 16. A compound capable of hybridizing to at least two exons in a pre-mRNA encoded by a gene, said compound comprising at least two parts wherein a first part comprises an oligonucleotide having at least 8 consecutive nucleotides that are complementary to a first of said at least two exons, and wherein a second part comprises an oligonucleotide having at least 8 consecutive nucleotides that are complementary to a second of said at least two exons in said pre-mRNA.
Specification