| 纯度 | >90%SDS-PAGE. |
| 种属 | Human |
| 靶点 | IDUA |
| Uniprot No | P35475 |
| 内毒素 | < 0.01EU/μg |
| 表达宿主 | E.coli |
| 表达区间 | 28-653aa |
| 氨基酸序列 | APHLVHVDAARALWPLRRFWRSTGFCPPLPHSQADQYVLSWDQQLNLAYVGAVPHRGIKQVRTHWLLELVTTRGSTGRGLSYNFTHLDGYLDLLRENQLLPGFELMGSASGHFTDFEDKQQVFEWKDLVSSLARRYIGRYGLAHVSKWNFETWNEPDHHDFDNVSMTMQGFLNYYDACSEGLRAASPALRLGGPGDSFHTPPRSPLSWGLLRHCHDGTNFFTGEAGVRLDYISLHRKGARSSISILEQEKVVAQQIRQLFPKFADTPIYNDEADPLVGWSLPQPWRADVTYAAMVVKVIAQHQNLLLANTTSAFPYALLSNDNAFLSYHPHPFAQRTLTARFQVNNTRPPHVQLLRKPVLTAMGLLALLDEEQLWAEVSQAGTVLDSNHTVGVLASAHRPQGPADAWRAAVLIYASDDTRAHPNRSVAVTLRLRGVPPGPGLVYVTRYLDNGLCSPDGEWRRLGRPVFPTAEQFRRMRAAEDPVAAAPRPLPAGGRLTLRPALRLPSLLLVHVCARPEKPPGQVTRLRALPLTQGQLVLVWSDEHVGSKCLWTYEIQFSQDGKAYTPVSRKPSTFNLFVFSPDTGAVSGSYRVRALDYWARPGPFSDPVPYLEVPVPRGPPSPGNP |
| 预测分子量 | 82.9 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. |
以下是关于IDUA重组蛋白的3条参考文献概览(基于公开研究整理)：
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1. **文献名称**: *Recombinant human α-L-iduronidase: Production, purification, and characterization*
**作者**: Kakkis ED 等
**摘要**: 研究报道了在CHO细胞中重组表达人源IDUA酶的方法，通过亲和层析纯化获得高活性酶，并在体外实验中证实其可有效降解Hurler综合征患者细胞中的糖胺聚糖底物，为酶替代疗法奠定基础。
2. **文献名称**: *Enzyme replacement therapy with laronidase improves outcomes in patients with mucopolysaccharidosis I: A phase III clinical trial*
**作者**: Muenzer J 等
**摘要**: 该III期临床试验评估了重组IDUA(商品名Aldurazyme®)治疗黏多糖贮积症I型患者的效果，结果显示患者尿液中糖胺聚糖水平显著下降，呼吸功能和生活质量改善，验证了长期治疗的安全性及有效性。
3. **文献名称**: *PEGylated recombinant human iduronidase (SA-ERU) exhibits extended plasma half-life and improved biodistribution in murine models*
**作者**: Giugliani R 等
**摘要**: 研究通过聚乙二醇(PEG)修饰IDUA重组蛋白，发现修饰后的酶在血浆半衰期延长了3倍，并在小鼠模型中显示出更优的组织分布特性，提示PEG化可能提升酶替代疗法的疗效。
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注：以上内容综合了公开领域的研究进展，具体文献信息需通过学术数据库(如PubMed)核实。如需实际引用，请确认作者、标题及期刊的准确性。
Recombinant IDUA (alpha-L-iduronidase) is a biologically engineered version of the human lysosomal enzyme encoded by the *IDUA* gene. This enzyme plays a critical role in the stepwise degradation of glycosaminoglycans (GAGs), specifically dermatan sulfate and heparan sulfate, by catalyzing the hydrolysis of alpha-L-iduronic acid residues. Deficiencies in IDUA activity lead to mucopolysaccharidosis type I (MPS I), a rare inherited lysosomal storage disorder characterized by the accumulation of undegraded GAGs in tissues and organs. MPS I manifests as a spectrum of severe clinical symptoms, including skeletal abnormalities, organomegaly, cardiorespiratory complications, and neurological decline.
The development of recombinant IDUA marked a breakthrough in enzyme replacement therapy (ERT) for MPS I. Produced via recombinant DNA technology in mammalian cell systems (e.g., Chinese hamster ovary cells), the protein is designed to mimic the structure and function of native human IDUA. Post-translational modifications, such as glycosylation, are critical for ensuring enzyme stability, cellular uptake via mannose-6-phosphate receptors, and lysosomal targeting. Laronidase (commercialized as Aldurazyme®) was the first FDA-approved recombinant IDUA therapy in 2003. demonstrating efficacy in reducing GAG storage and improving somatic symptoms, though it cannot cross the blood-brain barrier to address neurological involvement.
Ongoing research focuses on optimizing recombinant IDUA formulations, including PEGylation for extended half-life, engineered variants with enhanced catalytic activity, and hybrid approaches combining ERT with gene therapy. These innovations aim to improve treatment outcomes, reduce infusion frequency, and address unmet needs in severe MPS I subtypes. Additionally, recombinant IDUA serves as a vital tool for studying disease mechanisms and screening potential small-molecule chaperones. Its clinical success has established a paradigm for developing enzyme therapies for other lysosomal storage disorders.
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