Nexiguran

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National Institutes of Health Divider Arrow

NCATS

Approval Year

Substance Class	Nucleic Acid Created by `admin` on `Wed Apr 02 14:23:24 GMT 2025` Edited by `admin` on `Wed Apr 02 14:23:24 GMT 2025`
Nucleic Acid Type	sgRNA
Record UNII	5G537B4BTJ
Record Status	`Validated (UNII)`
Record Version

Download

Name

Type

Language

NTLA-2001 component hu-G000480

Preferred Name

English

Nexiguran

Official Name

English

NEXIGURAN [USAN]

Common Name

English

nexiguran [INN]

Common Name

English

RNA (synthetic human transthyretin gene TTR-specific single guide G009979)

Common Name

English

all-P-ambo-2'-O-methyl-P-thioadenylyl-(3'?5')-2'-O-methyl-P-thioadenylyl-(3'?5')-2'-O-methyl-P-thioadenylyl-(3'?5')-guanylyl-(3'?5')-guanylyl-(3'?5')-cytidylyl-(3'?5')-uridylyl-(3'?5')-guanylyl-(3'?5')-citidylyl-(3'?5')-uridylyl-(3'?5')-guanylyl-(3'?5')-

Common Name

English

single-stranded guide RNA (sgRNA) targeting the human transthyretin (TTR) gene with its 5'-terminal 20 nucleotides

Common Name

English

RNA (synthetic human transthyretin gene TTR-specific single guide)

Common Name

English

Code System Code Type Description

CAS

2305428-07-1

Created by admin on Wed Apr 02 14:23:24 GMT 2025 , Edited by admin on Wed Apr 02 14:23:24 GMT 2025

PRIMARY

SMS_ID

300000050066

Created by admin on Wed Apr 02 14:23:24 GMT 2025 , Edited by admin on Wed Apr 02 14:23:24 GMT 2025

PRIMARY

NCI_THESAURUS

C190369

Created by admin on Wed Apr 02 14:23:24 GMT 2025 , Edited by admin on Wed Apr 02 14:23:24 GMT 2025

PRIMARY

FDA UNII

5G537B4BTJ

Created by admin on Wed Apr 02 14:23:24 GMT 2025 , Edited by admin on Wed Apr 02 14:23:24 GMT 2025

PRIMARY

CAS

2644621-84-9

Created by admin on Wed Apr 02 14:23:24 GMT 2025 , Edited by admin on Wed Apr 02 14:23:24 GMT 2025

ALTERNATIVE

USAN

MN-130

Created by admin on Wed Apr 02 14:23:24 GMT 2025 , Edited by admin on Wed Apr 02 14:23:24 GMT 2025

PRIMARY

INN

12329

Created by admin on Wed Apr 02 14:23:24 GMT 2025 , Edited by admin on Wed Apr 02 14:23:24 GMT 2025

PRIMARY

Sugar	Site Range	Site Count
2'-O-Methyl-Ribose	1_1-1_3;1_29-1_40;1_69-1_100	47 / 100
D-Ribose (RNA)	1_4-1_28;1_41-1_68	53 / 100

Linkage	Site Range	Site Count
Phosporothioate	1_2-1_4;1_98-1_100	6 / 99
Phosphate	1_5-1_97	93 / 99

Related Record Type Details


					5G537B4BTJ

Nexiguran

ACTIVE MOIETY

Comments:

single-stranded guide RNA (sgRNA) targeting the human transthyretin (TTR) gene

Amount: