4. Wilsey M, Crawford BE, Schweighardt B, Cook JW, Wechsler T, Mueller WF.AAV9-NGLY1 gene replacement therapy improves phenotypic and biomarker endpoints in a rat model of NGLY1 Deficiency.Mol Ther Methods Clin Dev. 2022 Oct 3;27:259-271. 5. Frater CH, Ruzhnikov MRZ, Beres S, A...
Grace Science Announces FDA Clearance of Investigational New Drug (IND) Application to Initiate a Phase 1/2/3 Trial for the Treatment of NGLY1 Deficiency with GS-100, an AAV9 NGLY1 Gene Therapy 译:黄娟
Gene ID: 55768 种属 Homo sapiens 基因序列编号: NM_018297.4 基因描述 Homo sapiens N-glycanase 1 (NGLY1), transcript variant 1, mRNA DNA编码区: atggcggcggcggcattgggcagctcctcaggctcggcgtccccggccgtggctgagctc tgccagaacaccccggagacctttttggaggcctccaagctgctgctcacctatgctgac aacatcctcagaaaccctaatgatgaaaaa...
提供的NGLY1人源重组蛋白(P01)产品为带有GST标签的人源NGLY1全长序列重组蛋白,其对应的GeneID号为55768。 产品描述 与其它公司提供的重组蛋白不同,该NGLY1人源全长重组蛋白(P01)产品为采用CFS的无细胞麦胚蛋白合成系统表达出来的重组蛋白,可表达出对细胞有毒性、易被蛋白酶降解的蛋白;并获得具有良好的可溶性,并...
protein-coding gene 同源简称(alias symbol) FLJ11005|PNG1 基因家族代码(gene_family_id) [HGNC][GenScript] omim代码 610661 entrez码 55768[Vega] ensembl基因码 ENSG00000151092[Ensembl] 基因家族(gene family) refseq数据库代号 NM_018297 更多... ...
All mutations were predicted to be disease causing with in-silico prediction tools, and affected at least one feature of gene splicing. Protein modeling showed missense variants may affect covalent bonding within the protein structure, or interrupt active/binding amino-acid residues. In vitro studies...
这种疾病的患者在生命早期就会出现衰弱性症状,包括全身发育迟缓、认知障碍、(低)乳糖、运动障碍和其他神经系统症状。 原文标题: Grace Science, LLC Announces First Patient Treated in Phase 1/2/3 Trial of GS-100, an AAV9 Gene Therapy for NGLY1 Deficiency...
a gene therapy candidate, is a recombinant, single-stranded adeno-associated virus (AAV) 9 vector designed to deliver a functional copy of the human NGLY1 gene. Using the Ngly1 -/- rat, we tested different administration routes for GS-100: intracerebroventricular (ICV), intravenous (IV), ...
Multiple transcript variants encoding different isoforms have been found for this gene.[provided by RefSeq, Feb 2009] Also known as: CDG1V; hPNGase; N glycanase 1; Anti-NGLY1抗体NGLY 1; Peptide N(4) (N acetyl beta glucosaminyl asparagine amidase; Peptide:N glycanase; PNG1; PNGase. ...
克隆类型 Polyclonal Antibody 亚型 IgG 交叉反应 Human ; Mouse ; Rat 预测交叉反应 Monkey;Chicken 应用 IHC 稀释比例 IHC 1:50-200. 抗体信息 靶标 NGLY1 别名 NGLY1;CDDG;CDG1V;PNG1;PNGase 背景说明 This gene encodes an enzyme that catalyzes hydrolysis of an N(4)-(acetyl-beta-D-glucosaminyl...