Non-coding rna-mediated neurological disease treatment
AssigneeShanghai Genemagic Biosciences Co., Ltd.
InventorsHaibo ZHOU, Xinde HU, Jinlin SU
Life of this patent
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Abstract
A method for producing neurons from non-neuronal cells is provided, and comprises: by means of enhancing or reducing the expression or activity of certain miRNAs and/or lncRNAs in non-neuronal cells, trans differentiating or reprogramming the non-neuronal cells into neurons. Also provided is the use of reagents that enhance or reduce the expression or activity of certain miRNAs and/or lncRNAs in the prevention and/or treatment of diseases related to neuronal dysfunction or death.
Claims30
A method of producing neuronal cells from non-neuronal cells, comprises transdifferentiation or reprogramming the non-neuronal cells into neuronal cells by enhancing the expression or activity of miRNA selected from Let-7a, Let-7b, miR-18a/b, miR-24-3p, miR-34a, miR-92b, miR-96, miR-106, miR-125a/b, miR-128, miR-134, miR-135, miR-137, miR-141, miR-143-3p, miR-184, miR-200, miR-218, miR-219, miR-228, miR-284, miR-429, miR-430, or enhancing the expression or activity of lncRNA selected from utNgn1, RMST, Tuna, Linc-Brn1b, Dali, Miat/Gomafu, NBAT-1, Malat1, Dlx1as, Six3os, Evf2, LncKdm2b, lncRNA_N1, ncRNA_N2, lncRNA_N3, or any combination thereof,
- preferably, the expression or activity of the miRNA or lncRNA is enhanced through, for example, overexpression, gene activators, epigenetic modifications, miRNA mimics, direct delivery of RNA, small-molecule compounds, and/or RNA stabilizers.
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A method for producing neuronal cells from non-neuronal cells, comprises transdifferentiation or reprogramming the non-neuronal cells into neuronal cells by reducing the expression or activity of miRNA selected from miR-7a, miR-15, miR-23a/b, miR-25, miR-29a, miR-129, miR-137, miR-138, miR-155, miR-195, miR-214, miR-222, miR-223, miR-132, miR-133, or reducing the expression or activity of lncRNA selected from Pnky, Paupar, HOTAIRM1, IncR492, TUG1, or any combination thereof,
- preferably, the expression or activity of the miRNA or lncRNA is reduced by techniques such as DNA editing or RNA editing induced by gene editing technology, RNA expression inhibitors, antisense oligonucleotides, small RNA interference, miRNA technology, small molecule compounds, genes inhibiting technology, and/or epigenetic regulation;
- more preferably, wherein RNA editing includes CRISPR-mediated RNA degradation or RNA translation inhibition, RNA single base editing, insertion or deletion of bases of RNA, alteration of RNA splicing, or RNA epigenetic modifications.
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Pharmaceutical composition or pharmaceutical kit or reagent kit, which comprises reagent that enhance the expression or activity of miRNA selected from Let-7a, Let-7b, miR-18a/b, miR-24-3p, miR-34a, miR-92b, miR-96, miR-106, miR-125a/b, miR-128, miR-134, miR-135, miR-137, miR-141, miR-143-3p, miR-184, miR-200, miR-218, miR-219, miR-228, miR-284, miR-429, miR-430, or lncRNA selected from utNgn1, RMST, Tuna, Linc-Brn1b, Dali, Miat/Gomafu, NBAT-1, Malat1, Dlx1as, Six3os, Evf2, LncKdm2b, lncRNA_N1, lncRNA_N2, lncRNA_N3 lncRNA, or reagent that enhance the expression or activity of any combination thereof;
- or a reagent that reduce the expression or activity of miRNA selected from miR-7a, miR-15, miR-23a/b, miR-25, miR-29a, miR-129, miR-137, miR-138, miR-155, miR-195, miR-214, miR-222, miR-223, miR-132, miR-133, or lncRNA selected from Pnky, Paupar, HOTAIRM1, IncR492, TUG1, or reagent that reduce the expression or activity of any combination thereof.
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Dependencies are inferred from the claim text.