Strategy for suppressing the expression of an endogeneous gene by using compounds that are able to bind to the non-coding regions of the gene to be suppressed
OwnerGwenyth Jane Farrar, Peter Humphries +1inventors
InventorsGwenyth Jane Farrar, Peter Humphries, Paul Francis Kenna
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Abstract
The invention provides strategy for suppressing expression of an endogenous gene, wherein said strategy comprises providing suppression effectors able to bind to the non-coding regions of a gene to be suppressed, to prevent the functional expression thereof. The suppression effectors may be antisense nucleic acids, and the non-coding regions can include the transcribed but non-translated regions of a gene. The strategy can also introduce a replacement gene.
Claims39
A therapeutic composition for treating a genetic disease, the composition comprising:
- a) a suppression effector binds to an untranslated region of a mature RNA encoding a mutant allele, wherein said suppression effector inhibits the expression of the mutant allele;
- and
- b) a replacement nucleic acid that encodes a wild-type or non-disease causing allele and that is not inhibited by the suppression effector.
A therapeutic composition for suppressing the expression of a mutant allele of a protein, the composition comprising:
- a) a ribozyme that targets an untranslated region of a mature RNA encoding a mutant allele;
- and
- b) a replacement nucleic acid that encodes a wild-type or non-disease causing allele and that is not targeted by the ribozyme.
Dependencies are inferred from the claim text.