| 纯度 | >90%SDS-PAGE. |
| 种属 | Human |
| 靶点 | PPT1 |
| Uniprot No | P50897 |
| 内毒素 | < 0.01EU/μg |
| 表达宿主 | E.coli |
| 表达区间 | 1-306aa |
| 氨基酸序列 | MASPGCLWLLAVALLPWTCASRALQHLDPPAPLPLVIWHGMGDSCCNPLS MGAIKKMVEKKIPGIYVLSLEIGKTLMEDVENSFFLNVNSQVTTVCQALA KDPKLQQGYNAMGFSQGGQFLRAVAQRCPSPPMINLISVGGQHQGVFGLP RCPGESSHICDFIRKTLNAGAYSKVVQERLVQAEYWHDPIKEDVYRNHSI FLADINQERGINESYKKNLMALKKFVMVKFLNDSIVDPVDSEWFGFYRSG QAKETIPLQETSLYTQDRLGLKEMDNAGQLVFLATEGDHLQLSEEWFYAH IIPFLG |
| 预测分子量 | 60 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PPT1重组蛋白的3篇参考文献及其摘要概括：
1. **《Expression and characterization of recombinant human PPT1 in CHO cells for neuronal ceroid lipofuscinosis therapy》**
- 作者：Zhang Y, et al.
- 摘要：研究利用CHO细胞成功表达具有活性的重组人PPT1蛋白，通过体外实验证实其能有效降解棕榈酰化底物，为神经元蜡样脂褐质沉积症(NCL)的酶替代疗法提供潜在治疗策略。
2. **《Purification and functional analysis of PPT1 variants: Implications for CLN1 disease pathogenesis》**
- 作者：Vesa J, et al.
- 摘要：报道了重组PPT1蛋白的纯化方法及酶动力学分析，发现疾病相关突变导致PPT1酶活性显著降低，揭示了CLN1患者溶酶体功能障碍的分子机制。
3. **《Intracerebral delivery of recombinant PPT1 prolongs survival in a mouse model of infantile Batten disease》**
- 作者：Sleat DE, et al.
- 摘要：通过小鼠模型验证重组PPT1蛋白的脑内递送效果，结果显示其能延缓神经退行性病变并延长寿命，支持酶替代疗法在婴儿型Batten病中的治疗潜力。
(注：以上文献为虚拟示例，实际引用需根据具体论文调整。)
**Background of PPT1 Recombinant Protein**
Palmitoyl-protein thioesterase 1 (PPT1) is a lysosomal enzyme encoded by the *CLN1* gene, playing a critical role in lipid metabolism by catalyzing the removal of palmitate groups from S-palmitoylated proteins. This process, termed depalmitoylation, is essential for maintaining cellular homeostasis, protein trafficking, and membrane dynamics. Mutations in *CLN1* disrupt PPT1 activity, leading to the accumulation of lipid-modified proteins in lysosomes. This dysfunction is directly linked to neuronal ceroid lipofuscinosis type 1 (CLN1 disease), a fatal neurodegenerative disorder classified under Batten disease. CLN1 typically manifests in infancy with rapid cognitive decline, vision loss, seizures, and premature death, highlighting the enzyme’s vital role in neuronal health.
Recombinant PPT1 protein is produced via genetic engineering, often expressed in mammalian cell lines or microbial systems to ensure proper post-translational modifications and functional fidelity. Its production enables detailed biochemical studies, including enzyme kinetics, substrate specificity, and structural analysis. Recombinant PPT1 also serves as a therapeutic candidate; enzyme replacement therapy (ERT) trials aim to deliver functional PPT1 to the central nervous system, though challenges like blood-brain barrier penetration persist. Additionally, it aids in drug screening for small-molecule activators or stabilizers of PPT1.
Research on PPT1 recombinant protein has broader implications for understanding lysosomal storage disorders and neurodegenerative mechanisms. Recent advances in gene therapy and targeted delivery systems, such as nanoparticle carriers or fusion proteins, further underscore its potential in treating CLN1 and related conditions. By bridging molecular insights with therapeutic innovation, PPT1 recombinant protein remains a pivotal tool in both basic science and clinical exploration.
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