| 纯度 | > 95 % SDS-PAGE. |
| 种属 | Human |
| 靶点 | GLB1 |
| Uniprot No | P16278 |
| 内毒素 | < 0.01EU/μg |
| 表达宿主 | E.coli |
| 表达区间 | 24-677aa |
| 氨基酸序列 | NATQRMFEIDYSRDSFLKDGQPFRYISGSIHYSRVPRFYWKDRLLKMKMA GLNAIQTYVPWNFHEPWPGQYQFSEDHDVEYFLRLAHELGLLVILRPGPY ICAEWEMGGLPAWLLEKESILLRSSDPDYLAAVDKWLGVLLPKMKPLLYQ NGGPVITVQVENEYGSYFACDFDYLRFLQKRFRHHLGDDVVLFTTDGAHK TFLKCGALQGLYTTVDFGTGSNITDAFLSQRKCEPKGPLINSEFYTGWLD HWGQPHSTIKTEAVASSLYDILARGASVNLYMFIGGTNFAYWNGANSPYA AQPTSYDYDAPLSEAGDLTEKYFALRNIIQKFEKVPEGPIPPSTPKFAYG KVTLEKLKTVGAALDILCPSGPIKSLYPLTFIQVKQHYGFVLYRTTLPQD CSNPAPLSSPLNGVHDRAYVAVDGIPQGVLERNNVITLNITGKAGATLDL LVENMGRVNYGAYINDFKGLVSNLTLSSNILTDWTIFPLDTEDAVRSHLG GWGHRDSGHHDEAWAHNSSNYTLPAFYMGNFSIPSGIPDLPQDTFIQFPG WTKGQVWINGFNLGRYWPARGPQLTLFVPQHILMTSAPNTITVLELEWAP CSSDDPELCAVTFVDRPVIGSSVTYDHPSKPVEKRLMPPPPQKNKDSWLD HVVDHHHHHH |
| 预测分子量 | 74 kDa |
| 蛋白标签 | His tag N-Terminus |
| 缓冲液 | PBS, pH7.4, containing 0.01% SKL, 1mM DTT, 5% Trehalose and Proclin300. |
| 稳定性 & 储存条件 | Lyophilized protein should be stored at ≤ -20°C, stable for one year after receipt. Reconstituted protein solution can be stored at 2-8°C for 2-7 days. Aliquots of reconstituted samples are stable at ≤ -20°C for 3 months. |
| 复溶 | Always centrifuge tubes before opening.Do not mix by vortex or pipetting. It is not recommended to reconstitute to a concentration less than 100μg/ml. Dissolve the lyophilized protein in distilled water. Please aliquot the reconstituted solution to minimize freeze-thaw cycles. |
1. **"Production and characterization of recombinant human lysosomal β-galactosidase in CHO cells"**
*Author: C. Wang et al.*
摘要：研究报道了在CHO细胞中重组表达人源GLB1蛋白的方法，通过优化培养条件提高酶产量，并验证其酶活性和溶酶体靶向性，为酶替代疗法提供基础。
2. **"Structural analysis of GLB1 mutants associated with GM1 gangliosidosis"**
*Author: M. Suzuki et al.*
摘要：通过杆状病毒系统表达多种GLB1突变体，结合晶体结构解析和酶动力学分析，揭示了致病突变对蛋白稳定性及催化功能的影响。
3. **"Development of a high-yield insect cell system for GLB1 production"**
*Author: J. Lee & R. Smith*
摘要：利用昆虫细胞-杆状病毒表达系统规模化生产重组GLB1.优化纯化流程后获得高纯度蛋白，体外实验证实其可有效降解GM1神经节苷脂底物。
4. **"GLB1 enzyme replacement therapy in a murine model of GM1 gangliosidosis"**
*Author: K. Chen et al.*
摘要：在小鼠模型中评估重组GLB1蛋白的治疗效果，结果显示静脉注射后酶可穿透血脑屏障，显著减少中枢神经系统糖脂蓄积，改善神经功能。
**Background of GLB1 Recombinant Protein**
GLB1 (β-galactosidase-1) is a lysosomal enzyme encoded by the *GLB1* gene, responsible for hydrolyzing terminal β-linked galactose residues in glycoconjugates, including gangliosides and keratan sulfate. This enzymatic activity is critical for maintaining lysosomal function and cellular homeostasis. Mutations in *GLB1* lead to two autosomal recessive lysosomal storage disorders: GM1 gangliosidosis (neurodegenerative) and Morquio B syndrome (skeletal dysplasia), both characterized by substrate accumulation, cellular dysfunction, and progressive tissue damage.
Recombinant GLB1 protein is produced via genetic engineering in heterologous systems (e.g., mammalian cells, yeast, or bacteria) to mimic the native enzyme's structure and function. Its development aims to address the limitations of natural enzyme sources, such as low yield and immunogenicity, while enabling scalable production for therapeutic and research applications. In therapeutics, recombinant GLB1 is explored for enzyme replacement therapy (ERT) to restore enzymatic activity in patients. Preclinical studies show promise in reducing substrate accumulation and ameliorating disease phenotypes, though challenges like blood-brain barrier penetration for neurological forms remain.
Beyond therapy, recombinant GLB1 serves as a tool for studying lysosomal biology, substrate specificity, and disease mechanisms. Structural analyses using recombinant protein have elucidated catalytic domains and mutation-induced functional impairments, guiding drug design. Additionally, it is used in gene therapy research, such as adeno-associated virus (AAV) vectors delivering *GLB1* to target tissues. Recent advances include engineered variants with enhanced stability or modified glycosylation patterns to improve efficacy.
Overall, GLB1 recombinant protein represents a pivotal resource for understanding lysosomal disorders and developing targeted interventions, bridging gaps between basic research and clinical translation.
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