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SMN1

SMN1 詳細

英語名	Survival motor neuron protein	市販薬剤の数	3
治験薬の数	9	ターゲットの別称	Survival Of Motor Neuron 1 Isoform D345，Survival Of Motor Neuron 1 Isoform D2A2B3457，Survival Of Motor Neuron 1 Isoform D347，SMN1 more，SMN1，Survival Of Motor Neuron 1 Isoform D3457，Survival Of Motor Neuron 1， Telomeric，Gemin-1，Tudor Domain Containing 16A，SMNT，SMN，Survival Motor Neuron 1 Protein，Survival Motor Neuron Protein，T-BCD541，TDRD16A，BCD541，GEMIN1，SMA1，SMA2，SMA3，SMA4，SMA@，SMA，SMN Complex Proteins，Component of gems 1，SMN2，SMNC，Survival Of Motor Neuron 1 Isoform D2B3457，Survival Of Motor Neuron 1 Isoform D2A3457，Spinal Muscular Atrophy (Werdnig-Hoffmann Disease， Kugelberg-Welander Disease)，Survival Of Motor Neuron 1 Isoform D2A2B345
研究＆開発段階	Approved

英語名	Survival motor neuron protein
ターゲットの別称	Survival Of Motor Neuron 1 Isoform D345，Survival Of Motor Neuron 1 Isoform D2A2B3457，Survival Of Motor Neuron 1 Isoform D347，SMN1 more，SMN1，Survival Of Motor Neuron 1 Isoform D3457，Survival Of Motor Neuron 1， Telomeric，Gemin-1，Tudor Domain Containing 16A，SMNT，SMN，Survival Motor Neuron 1 Protein，Survival Motor Neuron Protein，T-BCD541，TDRD16A，BCD541，GEMIN1，SMA1，SMA2，SMA3，SMA4，SMA@，SMA，SMN Complex Proteins，Component of gems 1，SMN2，SMNC，Survival Of Motor Neuron 1 Isoform D2B3457，Survival Of Motor Neuron 1 Isoform D2A3457，Spinal Muscular Atrophy (Werdnig-Hoffmann Disease， Kugelberg-Welander Disease)，Survival Of Motor Neuron 1 Isoform D2A2B345
市販薬剤の数	3
治験薬の数	9
研究＆開発段階	Approved

SMN1 製品リスト

属性
Protein (1)
製品ライブラリ
オンライン (1)
種
Human (1)
タグ
His Tag (1)
マーカー
Unconjugated (1)

製品番号	種	発現システム	製品説明	タンパク質の構造	純度	特性
SM1-H5145	Human	E. coli	Human SMN1 Protein, His Tag

総数量1

SMN1バックグランド

SMN1 is produced chiefly by the SMN1 gene, located on the telomeric portion of chromosome 5q. SMN1 protein is one of a group of proteins called the SMN complex, which is important for the maintenance of specialized nerve cells called motor neurons. In cells, the SMN complex plays an important role in processing mRNA. The SMN complex helps to assemble the cellular machinery needed to process pre-mRNA. The SMN complex is also important for the development of specialized outgrowths from nerve cells called dendrites and axons. Deletion or mutation of the SMN1 gene results in a reduced level of full-length SMN protein and manifests as a range of neuromuscular phenotypes in humans as the disease spinal muscular atrophy (SMA).

SMN1分子の別称

Survival Motor Neuron 1,SMN1

治験薬に関する情報

英語名	研究コード	研究開発ステージ	会社	適応症	臨床試験
vesemnogene lantuparvovec	AAV-hSMN1	Phase 3 Clinical	Lantu Biopharma Inc	Muscular Atrophy, Spinal	詳細
Salanersen	BIIB-115; ION-306	Phase 3 Clinical	Ionis Pharmaceuticals Inc	Muscular Atrophy, Spinal	詳細
Branaplam	LMI-070; NVP-LMI070-AAA; NVP-LMI070-NX; NVS-SM1	Phase 2 Clinical	Novartis Pharma Ag	Huntington Disease; Muscular Atrophy, Spinal	詳細
GB-221 (GEMMA Biotherapeutics)	GB-221	Phase 2 Clinical	Gemma Biotherapeutics Inc	Spinal Muscular Atrophies of Childhood	詳細
ANB-004	ANB-004	Phase 2 Clinical	BIOCAD JSC	Muscular Atrophy, Spinal	詳細
EXG001-307	EXG001-307	Phase 2 Clinical	Exegenesis Bio Inc	Muscular Atrophy, Spinal	詳細
SMN-C3	SMN-C3	Phase 1 Clinical	F. Hoffmann-La Roche Ltd, Ptc Therapeutics Inc	Muscular Atrophy, Spinal	詳細
SKG-0201	SKG-0201; SKG0201	Phase 1 Clinical	Skyline Therapeutics (Hong Kong) Ltd	Muscular Atrophy, Spinal; Spinal Muscular Atrophies of Childhood	詳細
GCB-001	GCB-001; GCB001	Clinical	Shanghai Jinke Bo Biotechnology Co Ltd	Spinal Muscular Atrophies of Childhood	詳細

市販薬剤に関する情報

英語名	研究コード	研究開発ステージ	会社	オリジナル製品名	最初に承認をした国	最初の適応症	最初に承認を受けた会社	承認された日付	適応症	臨床試験
Risdiplam	RG-7916; RO-7034067	Approved	Ptc Therapeutics Inc	EVRYSDI, 艾满欣	United States	Muscular Atrophy, Spinal	Genentech Inc	2020-08-07	Muscular Atrophy, Spinal	詳細
Nusinersen sodium	BIIB-058; ISIS-396443	Approved	Ionis Pharmaceuticals Inc	Spinraza	United States	Muscular Atrophy, Spinal	Biogen Idec	2016-12-23	Muscular Atrophy, Spinal	詳細
Onasemnogene abeparvovec	AVXS-101; scAAV-9.CB.SMN; OAV101; rAAV-9	Approved	Novartis Gene Therapies Inc, Novartis Pharma Ag	Zolgensma, Itvisma	United States	Muscular Atrophy, Spinal	Novartis Gene Therapies Inc	2019-05-24	Muscular Atrophy, Spinal; Spinal Muscular Atrophies of Childhood	詳細