| 纯度 | >90%SDS-PAGE. |
| 种属 | Human |
| 靶点 | SGSH |
| Uniprot No | P51688 |
| 内毒素 | < 0.01EU/μg |
| 表达宿主 | E.coli |
| 表达区间 | 1-502aa |
| 氨基酸序列 | MSCPVPACCALLLVLGLCRARPRNALLLLADDGGFESGAYNNSAIATPHL DALARRSLLFRNAFTSVSSCSPSRASLLTGLPQHQNGMYGLHQDVHHFNS FDKVRSLPLLLSQAGVRTGIIGKKHVGPETVYPFDFAYTEENGSVLQVGR NITRIKLLVRKFLQTQDDRPFFLYVAFHDPHRCGHSQPQYGTFCEKFGNG ESGMGRIPDWTPQAYDPLDVLVPYFVPNTPAARADLAAQYTTVGRMDQGV GLVLQELRDAGVLNDTLVIFTSDNGIPFPSGRTNLYWPGTAEPLLVSSPE HPKRWGQVSEAYVSLLDLTPTILDWFSIPYPSYAIFGSKTIHLTGRSLLP ALEAEPLWATVFGSQSHHEVTMSYPMRSVQHRHFRLVHNLNFKMPFPIDQ DFYVSPTFQDLLNRTTAGQPTGWYKDLRHYYYRARWELYDRSRDPHETQN LATDPRFAQLLEMLRDQLAKWQWETHDPWVCAPDGVLEEKLSPQCQPLHN EL |
| 预测分子量 | 81 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. |
以下是3-4条关于SGSH(硫酸酯酶修饰因子)重组蛋白的参考文献示例及摘要概括(部分信息为模拟生成，仅供参考)：
1. **文献名称**: "Expression and characterization of recombinant human sulfamidase in CHO cells"
**作者**: Bielicki, J., et al.
**摘要**: 研究团队通过CHO细胞表达系统成功重组表达了人源SGSH蛋白，并验证其酶活性。结果显示重组蛋白能有效降解硫酸乙酰肝素(HS)，为Sanfilippo综合征A型的酶替代疗法提供实验基础。
2. **文献名称**: "Enzyme replacement therapy for mucopolysaccharidosis IIIA using recombinant sulfamidase in murine models"
**作者**: Zhao, H., et al.
**摘要**: 该研究利用HEK293细胞生产重组SGSH蛋白，并在Sanfilippo A型小鼠模型中验证其疗效。结果显示静脉注射重组蛋白可部分恢复脑组织中的酶活性并减少底物积累，但存在血脑屏障穿透性挑战。
3. **文献名称**: "Optimization of sulfamidase production in E. coli for enhanced stability and activity"
**作者**: Khan, S., et al.
**摘要**: 通过大肠杆菌表达系统优化重组SGSH的生产工艺，引入分子伴侣共表达策略以提高蛋白折叠效率。纯化后的重组酶在体外实验中展现出与天然酶相当的活性，且热稳定性显著提升。
4. **文献名称**: "Gene therapy delivery of recombinant SGSH via AAV vectors in a canine model of MPS IIIA"
**作者**: Chen, Y., et al.
**摘要**: 采用AAV载体递送重组SGSH基因至Sanfilippo A型犬模型的中枢神经系统，结果显示长期稳定的酶活性表达，并显著改善神经退行性病变，为临床基因治疗提供依据。
(注：以上文献信息为模拟生成，实际研究中请通过PubMed/Google Scholar等平台以关键词"SGSH recombinant protein"或"sulfamidase gene therapy"检索最新文献。)
Sulfamidase (SGSH), also known as N-sulfoglucosamine sulfohydrolase, is a lysosomal enzyme critical for the stepwise degradation of heparan sulfate, a glycosaminoglycan (GAG). It specifically cleaves sulfate groups from N-sulfoglucosamine residues in heparan sulfate chains. Genetic mutations in the SGSH gene lead to deficient enzyme activity, resulting in the accumulation of undegraded heparan sulfate in lysosomes. This underlies mucopolysaccharidosis type IIIA (MPS IIIA or Sanfilippo syndrome type A), a rare autosomal recessive neurodegenerative disorder characterized by progressive cognitive decline, behavioral abnormalities, and shortened lifespan. Currently, no approved disease-modifying therapies exist for MPS IIIA.
Recombinant SGSH protein has been investigated as a potential therapeutic agent, particularly for enzyme replacement therapy (ERT). Production typically involves expressing the human SGSH gene in mammalian cell systems (e.g., CHO or HEK293 cells) to ensure proper post-translational modifications, including glycosylation essential for enzyme stability and lysosomal targeting. The recombinant protein is purified through chromatographic methods and rigorously characterized for activity, purity, and structure using techniques like SDS-PAGE, Western blotting, and mass spectrometry.
Preclinical studies in animal models have demonstrated that intravenously administered recombinant SGSH can reduce heparan sulfate storage in peripheral tissues. However, treating CNS manifestations remains challenging due to the enzyme's limited blood-brain barrier penetration. Innovative approaches being explored include intrathecal delivery, fusion proteins with brain-targeting peptides, or gene therapy vectors. Additionally, immune responses to the exogenous enzyme and high production costs present translational hurdles. Despite these challenges, recombinant SGSH represents a cornerstone strategy for developing biologics to address the root cause of MPS IIIA, with ongoing research aiming to optimize delivery and efficacy.
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