Method for treating usher syndrome and composition thereof

A LEAPER technology-based method for editing target RNA containing a G to A mutation in a USH2A gene transcript, comprising: introducing adenosine deaminase-recruiting RNA (arRNA) used to edit target RNA or a construct that encodes the arRNA into a cell; the arRNA comprises a complementary RNA seque...

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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 SPIRITS VINEGAR WINE
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Beschreibung
Zusammenfassung:	A LEAPER technology-based method for editing target RNA containing a G to A mutation in a USH2A gene transcript, comprising: introducing adenosine deaminase-recruiting RNA (arRNA) used to edit target RNA or a construct that encodes the arRNA into a cell; the arRNA comprises a complementary RNA sequence that hybridizes with the target RNA; and the arRNA can recruit adenosine deaminase (ADAR) that acts on the RNA, such that target adenosine in the target RNA is deaminated, safely and effectively carrying out in vivo editing of bases from A to I on RNA, repairing a pathogenic mutation site, and achieving the objective of treating diseases such as Usher syndrome.