Method and drug for treating Hurler syndrome

Disclosed are a LEAPER technique-based nucleic acid drug and a method for treating diseases such as Hurler syndrome by means of using the nucleic acid drug to target and edit RNA. The method comprises: using the nucleic acid drug to edit RNA from an adenosine base to a hypoxanthine base; precisely r...

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Bibliographische Detailangaben
Hauptverfasser:	ZHAO, Yanxia, TANG, Gangbin, YI, Zexuan, YUAN, Pengfei, LIU, Nengyin
Format:	Patent
Sprache:	eng
Schlagworte:	BEER BIOCHEMISTRY CHEMISTRY COMPOSITIONS THEREOF CULTURE MEDIA ENZYMOLOGY HUMAN NECESSITIES HYGIENE MEDICAL OR VETERINARY SCIENCE METALLURGY MICROBIOLOGY MICROORGANISMS OR ENZYMES MUTATION OR GENETIC ENGINEERING PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS SPIRITS VINEGAR WINE
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Beschreibung
Zusammenfassung:	Disclosed are a LEAPER technique-based nucleic acid drug and a method for treating diseases such as Hurler syndrome by means of using the nucleic acid drug to target and edit RNA. The method comprises: using the nucleic acid drug to edit RNA from an adenosine base to a hypoxanthine base; precisely repairing the pathogenic G>A mutation site of, for example, Hurler syndrome; and recovering the normal in vivo expression of a protein encoded by RNA, such as IDUA.