Gene correction induced neural stem cells using huntingtin gene-specific guide rna, and cell therapeutic agent using the same
AssigneeKorea University Research and Business Foundation
InventorsSunghoi HONG, Yong NAM-KUNG, Donghun HYUN
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Abstract
The present invention relates to: a gene editing composition comprising a guide RNA for editing mutated CAG repeat sequences of neural stem cells (ciNSCs) induced from somatic cells of a Huntington's disease patient; the edited ciNSCs; and a cell therapeutic agent for Huntington's disease, comprising same, wherein, in the edited ciNSCs, Huntington gene mutations are removed by the guide RNA of the present invention, and thus treatment for Huntington's disease and neuronal proliferation can be improved.
Claims17
A composition for correcting a huntingtin (HTT) gene, comprising a guide RNA comprising a nucleotide sequence complementary to the 5′ untranslated region (UTR) and first exon of the huntingtin (HTT) gene, or DNA encoding the guide RNA;
- and
- a Cas protein or a polynucleotide encoding the same.
A method for correcting a huntingtin gene, the method comprising transducing DNA encoding a guide RNA including a nucleotide sequence complementary to the 5′ untranslated region (UTR) and first exon of the huntingtin (HTT) gene;
- and a polynucleotide encoding a Cas protein into induced neural stem cells (HD-ciNSCs) comprising a mutated huntingtin gene comprising an abnormal CAG repeat sequence.
A method for correcting a huntingtin gene, the method comprising transducing DNA encoding a guide RNA including a nucleotide sequence complementary to the 5′ untranslated region (UTR) and first exon of the huntingtin (HTT) gene;
- and a polynucleotide encoding a Cas protein into induced neural stem cells (HD-ciNSCs) comprising a mutated huntingtin gene comprising an abnormal CAG repeat sequence to correct the huntingtin gene.
Dependencies are inferred from the claim text.