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US 2026/0109985 A1Published application
Therapy by trans-splicing of opa1 pre-messenger rnas for the treatment of diseases associated with opa1 gene mutations
AssigneesCentre National De La Recherche Scientifique, Institut National de la Sante et de la Recherche Medicale, Universite D'Angers
InventorsYannick LE DANTEC, Guy LENAERS, Olivier BARIS, Arnaud CHEVROLLIER, Salim KHIATI
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Abstract
The present invention provides pre-mRNA trans-splicing molecules (RTMs) that are useful for correcting mutations in the OPA1 gene. Also provided are methods of using the RTMs as gene therapy (e.g., ex vivo and in vivo gene therapy) for the treatment or prevention of diseases or disorders associated with OPA1 mutations.
Claims27
A nucleic acid OPA1 pre-mRNA trans-splicing molecule comprising, operatively linked in a 5′-to-3′ direction or a 3′-to-5′ direction:
- (a) a binding domain configured to bind to an OPA1 target sequence comprising an intron, an intron/exon junction or an exon;
- (b) a splicing domain configured to mediate trans-splicing;
- and
- (c) a coding domain comprising a functional OPA1 exon,
- wherein the nucleic acid OPA1 pre-mRNA trans-splicing molecule is configured to trans-splice the coding domain to an endogenous mutated OPA1 exon adjacent to the OPA1 target sequence, thereby replacing the endogenous mutated OPA1 exon with the functional OPA1 exon and correcting a mutation in OPA1 transcripts.
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Dependencies are inferred from the claim text.