Patent classifications
C
CHEMISTRY; METALLURGY
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C12
BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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C12N
MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
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2501/00
Active agents used in cell culture processes, e.g. differentation
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C12N2501/80
Neurotransmitters; Neurohormones
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C12N2501/845
Gamma amino butyric acid [GABA]
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C12N2501/845

GENE CORRECTION INDUCED NEURAL STEM CELLS USING HUNTINGTIN GENE-SPECIFIC GUIDE RNA, AND CELL THERAPEUTIC AGENT USING THE SAME
20260115318 · 2026-04-30 · ·

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.