| 纯度 | >90%SDS-PAGE. |
| 种属 | Human |
| 靶点 | LIPA |
| Uniprot No | P38571 |
| 内毒素 | < 0.01EU/μg |
| 表达宿主 | E.coli |
| 表达区间 | 22-399aa |
| 氨基酸序列 | SGGKLTAVDPETNMNVSEIISYWGFPSEEYLVETEDGYILCLNRIPHGRKNHSDKGPKPVVFLQHGLLADSSNWVTNLANSSLGFILADAGFDVWMGNSRGNTWSRKHKTLSVSQDEFWAFSYDEMAKYDLPASINFILNKTGQEQVYYVGHSQGTTIGFIAFSQIPELAKRIKMFFALGPVASVAFCTSPMAKLGRLPDHLIKDLFGDKEFLPQSAFLKWLGTHVCTHVILKELCGNLCFLLCGFNERNLNMSRVDVYTTHSPAGTSVQNMLHWSQAVKFQKFQAFDWGSSAKNYFHYNQSYPPTYNVKDMLVPTAVWSGGHDWLADVYDVNILLTQITNLVFHESIPEWEHLDFIWGLDAPWRLYNKIINLMRKYQ |
| 预测分子量 | 47.1 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LIPA(溶酶体酸性脂肪酶)重组蛋白的3篇参考文献，简要概括作者、文献名称及摘要内容：
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1. **文献名称**: *"Sebelipase Alfa in Lysosomal Acid Lipase Deficiency: A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study"*
**作者**: Valayannopoulos V. et al.
**摘要**: 该3期临床试验评估重组LIPA酶(sebelipase alfa)治疗LAL缺乏症的安全性和有效性。结果显示，每周输注sebelipase alfa显著降低患者肝脏脂肪堆积并改善肝功能，证实其作为酶替代疗法的潜力。
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2. **文献名称**: *"Recombinant lysosomal acid lipase corrects lipid storage in murine and human models of LAL deficiency"*
**作者**: Du H. et al.
**摘要**: 研究通过体外细胞模型和LAL缺陷小鼠验证重组LIPA蛋白的功能，证明其能有效降解溶酶体内积累的胆固醇酯和三酰甘油，为LAL缺乏症的治疗提供临床前依据。
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3. **文献名称**: *"Production and characterization of recombinant human lysosomal acid lipase in mammalian cell systems"*
**作者**: Burke D.G. et al.
**摘要**: 文章优化了在哺乳动物细胞(CHO细胞)中重组表达人LIPA的工艺，分析了产物的酶活性、糖基化修饰及稳定性，为大规模生产高质量治疗用LIPA奠定基础。
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如需具体文献年份或补充DOI，可进一步提供关键词检索协助。
**Background of LIPA Recombinant Protein**
Lysosomal acid lipase (LIPA), encoded by the *LIPA* gene, is a critical enzyme responsible for hydrolyzing cholesterol esters and triglycerides within lysosomes. This process is essential for lipid metabolism and cellular homeostasis. Mutations in the *LIPA* gene lead to deficient or dysfunctional enzyme activity, resulting in rare autosomal recessive disorders such as Wolman disease (severe infantile form) and cholesteryl ester storage disease (CESD, a milder late-onset form). These conditions are characterized by progressive accumulation of lipid droplets in lysosomes, leading to multi-organ damage, liver dysfunction, and cardiovascular complications.
Recombinant LIPA protein has been developed to address the underlying enzyme deficiency in these disorders. Produced through genetic engineering in mammalian or microbial expression systems (e.g., Chinese hamster ovary cells or yeast), recombinant LIPA mimics the natural enzyme’s function, enabling targeted enzyme replacement therapy (ERT). For instance, sebelipase alfa (Kanuma®), a recombinant human LIPA approved by the FDA in 2015. has demonstrated efficacy in improving survival and reducing lipid accumulation in patients.
Beyond therapeutic applications, LIPA recombinant protein serves as a vital tool in biochemical research. It aids in studying lysosomal storage disorders, lipid metabolism pathways, and drug screening. Its high purity and activity ensure reproducibility in experiments, while engineered variants help explore structure-function relationships or enhance stability for industrial use.
Overall, recombinant LIPA protein bridges clinical and research needs, offering life-saving treatments for genetic lipid disorders and advancing our understanding of lysosomal biology.
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