Selbstspaltende Therapeutische Ribozym-Nukleinsäuremoleküle

Details

Veröffentlichungs-Nr.

EP4756023

Anmeldetag

6. Dezember 2024

Veröffentlichung

10. Juni 2026

Rechtsraum

EP

IPC

C12N15/113, C12N15/67

Abstract

The present invention relates to a therapeutic nucleic acid molecule comprising or consisting of an open reading frame comprising (i) a part of a gene, wherein the part comprises (a) at least one intron and the flanking exons of the gene, (b) at least two exons of the gene, wherein the first exon encodes the 5'UTR or the last exon encodes the 3'UTR, (c) at least one exon of the gene preceded by an exogenous 5'UTR or followed by an exogenous 3'UTR, or (d) at least one exon of the gene, wherein the gene is associated with a disease in humans or is useful for treating a disease in humans, wherein the disease can be treated by the expression of the gene itself or one of its functional or sequence paralogs; and (ii) inserted within the part of a gene according to (i) a nucleic acid sequence encoding a self-cleavable ribozyme, wherein (a') in (a) the nucleic acid sequence encoding a self-cleavable ribozyme is inserted into the intron, (b') in (b) the nucleic acid sequence encoding a self-cleavable ribozyme is inserted into the 5' or 3'UTR, (c') in (c) the nucleic acid sequence encoding a self-cleavable ribozyme is inserted into the exogenous 5' or 3'UTR, and (d') in (d) the nucleic acid sequence encoding a self-cleavable ribozyme is inserted into at least one exon.

Anmelder

Firma

Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V.

Land

🇩🇪 Deutschland