Pasemnogene gleparvovec transgene insert encoding codon-optimised human survival of motor neuron 1 (SMN1) gene

Approval Year

Substance Class	Nucleic Acid Created by `admin` on `Wed Apr 02 19:51:09 GMT 2025` Edited by `admin` on `Wed Apr 02 19:51:09 GMT 2025`
Nucleic Acid Type	VECTOR
Sequence Origin	VIRAL, HUMAN
Sequence Type	COMPLETE
Record UNII	JZW2TY9A6W
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

Pasemnogene gleparvovec transgene insert encoding codon-optimised human survival of motor neuron 1 (SMN1) gene

Common Name

English

DNA (recombinant adeno-associated virus vector pAAV-SMN1-GeneBeam v2020-03-11 cytomegalovirus enhancer region plus cytomegalovirus promoter region plus human ?-globin gene intron fragment plus human survival of motor neuron protein SMN1 codon-optimized g

Preferred Name

English

Code System Code Type Description

CAS

2609078-43-3

Created by admin on Wed Apr 02 19:51:09 GMT 2025 , Edited by admin on Wed Apr 02 19:51:09 GMT 2025

PRIMARY

FDA UNII

JZW2TY9A6W

Created by admin on Wed Apr 02 19:51:09 GMT 2025 , Edited by admin on Wed Apr 02 19:51:09 GMT 2025

PRIMARY

Sugar	Site Range	Site Count
Deoxyribose (DNA)	1_1-1_2924	2924 / 2924

Linkage	Site Range	Site Count
Phosphate	1_2-1_2924	2923 / 2923