ASGR2 (asialoglycoprotein receptor 2) is a transmembrane protein belonging to the C-type lectin superfamily, predominantly expressed on the surface of hepatocytes. It plays a critical role in glycoprotein homeostasis by recognizing and binding to desialylated (galactose-terminal) glycoproteins, triggering their endocytosis and lysosomal degradation. This receptor forms a heteromeric complex with ASGR1. enhancing ligand-binding specificity and cellular uptake efficiency. ASGR2 has garnered significant interest in biomedical research due to its liver-specific expression and potential as a therapeutic target or drug delivery vehicle.
Recombinant ASGR2 proteins are engineered to study its structural and functional properties, often produced in mammalian expression systems (e.g., HEK293 or CHO cells) to ensure proper post-translational modifications. These proteins typically include extracellular domains responsible for ligand interaction, fused with tags like Fc or His for purification and detection. Researchers utilize recombinant ASGR2 to investigate receptor-ligand interactions, screen targeted therapies, and develop liver-directed delivery systems for genes, drugs, or nanoparticles.
In clinical contexts, ASGR2 has been explored as a biomarker for liver diseases, including hepatocellular carcinoma and fibrosis. Its ability to mediate targeted internalization makes it valuable for designing ASGR2-binding ligands or antibodies in cancer therapy and antiviral strategies. Recent studies also examine its role in cholesterol metabolism regulation, potentially linking it to cardiovascular diseases. The development of recombinant ASGR2 continues to advance both basic research and translational applications in hepatology and precision medicine.
以下是3篇关于ASL(精氨酸琥珀酸裂解酶)重组蛋白的示例参考文献(内容为模拟概括，仅供参考)：
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1. **标题**: *Expression and Purification of Recombinant Human Argininosuccinate Lyase in E. coli for Enzyme Deficiency Therapy*
**作者**: Smith A, et al.
**摘要**: 本研究报道了通过大肠杆菌表达系统高效表达人源ASL重组蛋白的工艺，优化了纯化步骤，获得高活性酶。体外实验证实其可恢复缺乏ASL的细胞模型的尿素循环功能，为精氨酸琥珀酸尿症的酶替代疗法奠定基础。
2. **标题**: *Structural Characterization of Recombinant ASL and Its Therapeutic Efficacy in a Murine Model*
**作者**: Chen L, et al.
**摘要**: 通过X射线晶体学解析了重组ASL的三维结构，并利用ASL缺陷小鼠模型验证其体内疗效。结果显示，重组ASL显著降低血浆氨水平，改善代谢指标，证明其作为基因治疗载体的潜力。
3. **标题**: *Stability and Pharmacokinetics of PEGylated Recombinant ASL in Preclinical Studies*
**作者**: Gupta R, et al.
**摘要**: 研究对聚乙二醇(PEG)修饰的重组ASL进行稳定性与药代动力学分析。PEG化延长了半衰期，并在大鼠模型中显示更持久的氨清除能力，提示其可减少临床给药频率。
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**注**：以上文献信息为模拟示例，实际引用需查询真实数据库(如PubMed)并核实原文内容。建议使用关键词 "recombinant argininosuccinate lyase" 或 "ASL gene therapy" 在学术平台检索最新研究。
ASL (Argininosuccinate lyase) is a critical enzyme in the urea cycle, responsible for catalyzing the cleavage of argininosuccinate into arginine and fumarate. This reaction is essential for nitrogen disposal and the biosynthesis of arginine, a semi-essential amino acid. Deficiencies in ASL activity due to genetic mutations lead to argininosuccinic aciduria, a rare autosomal recessive disorder characterized by hyperammonemia, metabolic imbalance, and neurological complications. Traditional approaches to study or supplement ASL rely on isolating the enzyme from natural sources, but low abundance and purification challenges limit scalability and therapeutic applications.
Recombinant ASL protein, produced via genetic engineering, offers a sustainable and efficient alternative. By cloning the ASL gene into expression systems like *E. coli*, yeast, or mammalian cells, researchers achieve high-yield production with consistent quality. This method enables precise control over protein structure and post-translational modifications, enhancing functional fidelity. Recombinant ASL is pivotal in enzyme replacement therapy (ERT) for genetic disorders, providing a purified, bioavailable form to restore metabolic pathways. It also serves as a tool for mechanistic studies, drug screening, and diagnostic assays to evaluate urea cycle dysfunction.
Recent advancements focus on optimizing expression systems to improve protein stability and reduce immunogenicity. PEGylation and nanoparticle encapsulation are explored to prolong circulation half-life in therapeutic contexts. Additionally, recombinant ASL supports the development of gene therapies by serving as a reference for correcting enzymatic activity in cellular models. Despite progress, challenges like tissue-specific delivery and cost-effective manufacturing remain. Overall, recombinant ASL exemplifies the intersection of biotechnology and precision medicine, addressing unmet needs in rare metabolic diseases while advancing biochemical research.
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