OPA1 antisense oligomers for treatment of conditions and diseases
AssigneeStoke Therapeutics, Inc.
InventorsIsabel Aznarez, Aditya Venkatesh, Gene Liau
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Abstract
Alternative splicing events in genes can lead to non-productive mRNA transcripts which in turn can lead to aberrant protein expression, and therapeutic agents which can target the alternative splicing events in genes can modulate the expression level of functional proteins in patients and/or inhibit aberrant protein expression. Such therapeutic agents can be used to treat a condition or disease caused by protein deficiency and/or mitochondrial function deficit.
Claims20
A method of modulating expression of an OPA1 protein in a cell having a pre-mRNA, wherein the pre-mRNA is transcribed from an OPA1 gene, and wherein the pre-mRNA comprises a coding exon, the method comprising:
- contacting an agent or a vector encoding the agent to the cell,
- whereby the agent promotes exclusion of the coding exon from the pre-mRNA in the cell, thereby increasing a level of a processed mRNA that is processed from the pre-mRNA in the cell;
- and that lacks the coding exon,
- wherein the agent comprises an antisense oligomer, and
- wherein the antisense oligomer comprises a sequence with at least 80% sequence identity to one of the sequences set forth in SEQ ID NOs:
- 227-242, 250, 280-283, 288, and 290-292.
Dependencies are inferred from the claim text.