| 纯度 | > 95 % SDS-PAGE. |
| 种属 | Human |
| 靶点 | ARSA |
| Uniprot No | P54150 |
| 内毒素 | < 0.01EU/μg |
| 表达宿主 | E.coli |
| 表达区间 | 19-507aa |
| 氨基酸序列 | RPPNIVLIFA DDLGYGDLGC YGHPSSTTPN LDQLAAGGLR FTDFYVPVSL CTPSRAALLT GRLPVRMGMY PGVLVPSSRG GLPLEEVTVA EVLAARGYLT GMAGKWHLGV GPEGAFLPPH QGFHRFLGIP YSHDQGPCQN LTCFPPATPC DGGCDQGLVP IPLLANLSVE AQPPWLPGLE ARYMAFAHDL MADAQRQDRP FFLYYASHHT HYPQFSGQSF AERSGRGPFG DSLMELDAAV GTLMTAIGDL GLLEETLVIF TADNGPETMR MSRGGCSGLL RCGKGTTYEG GVREPALAFW PGHIAPGVTH ELASSLDLLP TLAALAGAPL PNVTLDGFDL SPLLLGTGKS PRQSLFFYPS YPDEVRGVFA VRSGKYKAHF FTQGSAHSDT TADPACHASS SLTAHEPPLL YDLSKDPGEN YNLLGGVAGA TPEVLQALKQ LQLLKAQLDA AVTFGPSQVA RGEDPALQIC CHPGCTPRPA CCHCPDPHAV DHHHHHH |
| 预测分子量 | 53 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ARSA(芳基硫酸酯酶A)重组蛋白的3篇代表性文献及其摘要概述：
1. **文献名称**：*Recombinant human arylsulfatase A: High-level expression in mouse milk and biochemical characterization*
**作者**：Matzner U, et al.
**摘要**：该研究报道了利用转基因小鼠乳腺生物反应器高效表达重组人ARSA蛋白，并证实其酶活性和稳定性与天然酶一致，为酶替代治疗提供了潜在生产途径。
2. **文献名称**：*Production of recombinant human arylsulfatase A in CHO cells: From clone screening to pharmacokinetics*
**作者**：Flores JA, et al.
**摘要**：通过优化CHO细胞表达系统规模化生产重组ARSA，详细描述了克隆筛选、纯化工艺及药代动力学特征，验证其在动物模型中的生物分布和代谢稳定性。
3. **文献名称**：*Structural and functional analysis of recombinant ARSA for metachromatic leukodystrophy therapy*
**作者**：Gieselmann V, et al.
**摘要**：解析了重组ARSA的晶体结构，通过体外细胞实验验证其纠正溶酶体贮积缺陷的能力，为改善异染性脑白质营养不良的酶替代疗法提供分子机制支持。
注：以上文献标题与作者为示例性内容，实际研究中需根据具体论文数据库(如PubMed)检索最新或关键文献。建议结合关键词“recombinant ARSA”、“arylsulfatase A expression”进一步查阅。
Arylsulfatase A (ARSA) is a lysosomal enzyme critical for the hydrolysis of sulfated glycolipids, particularly sulfatides, through the cleavage of sulfate groups. Deficiencies in ARSA activity due to genetic mutations lead to metachromatic leukodystrophy (MLD), a rare autosomal recessive neurodegenerative disorder characterized by sulfatide accumulation in the nervous system and peripheral organs. This results in progressive demyelination, motor and cognitive decline, and early mortality. Historically, MLD treatment options were limited to palliative care, but advances in recombinant protein technology have opened pathways for enzyme replacement therapy (ERT) and gene therapy.
Recombinant ARSA (rhARSA) is produced using mammalian cell expression systems, such as Chinese hamster ovary (CHO) or human embryonic kidney (HEK293) cells, to ensure proper post-translational modifications, including glycosylation, which enhances enzyme stability and lysosomal targeting. Preclinical studies demonstrated that intravenously administered rhARSA can reduce sulfatide storage in MLD model mice, though its efficacy in crossing the blood-brain barrier (BBB) remains limited. To address this, strategies like intrathecal delivery or fusion with BBB-penetrating peptides are being explored.
Clinical trials of ERT using rhARSA have shown partial success in alleviating peripheral symptoms but limited impact on central nervous system pathology. This has spurred interest in combinatorial approaches, such as coupling ERT with gene therapy using viral vectors (e.g., lentivirus or AAV) to deliver functional ARSA genes directly to neural cells. Additionally, recombinant ARSA serves as a vital tool for studying sulfatide metabolism and screening potential small-molecule chaperones for MLD. Despite challenges in delivery and immunogenicity, rhARSA remains a cornerstone in developing targeted therapies for MLD and related lysosomal storage disorders.
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