SMN1

SMN1

SMN1 詳細

Molecule Name
Survival motor neuron protein
分子の別称
Survival Motor Neuron 1,SMN1
Drug Development Progress
治験薬の数9
市販薬剤の数3
研究&開発段階Approved
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
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 製品リスト

Species
  • Human(1)
Tag
  • His Tag(1)
Host
  • E. coli(1)
Conjugate
  • Unconjugated(1)
Grade
  • RUO(1)
Low Endotoxin
  • No(1)
MoleculeCat. No.Product Name種HostStructurePurityActivitySize&Price
SMN1
Human SMN1 Protein, His Tag
Human
E. coli
SDS-PAGE
ELISA

$310.00

100ug
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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 lantuparvovecAAV-hSMN1Phase 3 ClinicalLantu Biopharma Inc
Muscular Atrophy, Spinal
詳細
SalanersenBIIB-115; ION-306Phase 3 ClinicalIonis Pharmaceuticals Inc
Muscular Atrophy, Spinal
詳細
BranaplamLMI-070; NVP-LMI070-AAA; NVP-LMI070-NX; NVS-SM1Phase 2 ClinicalNovartis Pharma Ag
Huntington Disease; Muscular Atrophy, Spinal
詳細
GB-221 (GEMMA Biotherapeutics)GB-221Phase 2 ClinicalGemma Biotherapeutics Inc
Spinal Muscular Atrophies of Childhood
詳細
ANB-004ANB-004Phase 2 ClinicalBIOCAD JSC
Muscular Atrophy, Spinal
詳細
EXG001-307EXG001-307Phase 2 ClinicalExegenesis Bio Inc
Muscular Atrophy, Spinal
詳細
SMN-C3SMN-C3Phase 1 ClinicalF. Hoffmann-La Roche Ltd, Ptc Therapeutics Inc
Muscular Atrophy, Spinal
詳細
SKG-0201SKG-0201; SKG0201Phase 1 ClinicalSkyline Therapeutics (Hong Kong) Ltd
Muscular Atrophy, Spinal; Spinal Muscular Atrophies of Childhood
詳細
GCB-001GCB-001; GCB001ClinicalShanghai 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
RisdiplamRG-7916; RO-7034067ApprovedPtc Therapeutics IncEVRYSDI, 艾满欣United StatesMuscular Atrophy, SpinalGenentech Inc2020-08-07
Muscular Atrophy, Spinal
詳細
Nusinersen sodiumBIIB-058; ISIS-396443ApprovedIonis Pharmaceuticals IncSpinrazaUnited StatesMuscular Atrophy, SpinalBiogen Idec2016-12-23
Muscular Atrophy, Spinal
詳細
Onasemnogene abeparvovecAVXS-101; scAAV-9.CB.SMN; OAV101; rAAV-9ApprovedNovartis Gene Therapies Inc, Novartis Pharma AgZolgensma, ItvismaUnited StatesMuscular Atrophy, SpinalNovartis Gene Therapies Inc2019-05-24
Muscular Atrophy, Spinal; Spinal Muscular Atrophies of Childhood
詳細
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