| 纯度 | >85%SDS-PAGE. |
| 种属 | Human |
| 靶点 | NAGLU |
| Uniprot No | P54802 |
| 内毒素 | < 0.01EU/μg |
| 表达宿主 | E.coli |
| 表达区间 | 24-743aa |
| 氨基酸序列 | DEAREAA AVRALVARLL GPGPAADFSV SVERALAAKP GLDTYSLGGG GAARVRVRGS TGVAAAAGLH RYLRDFCGCH VAWSGSQLRL PRPLPAVPGE LTEATPNRYR YYQNVCTQSY SFVWWDWARW EREIDWMALN GINLALAWSG QEAIWQRVYL ALGLTQAEIN EFFTGPAFLA WGRMGNLHTW DGPLPPSWHI KQLYLQHRVL DQMRSFGMTP VLPAFAGHVP EAVTRVFPQV NVTKMGSWGH FNCSYSCSFL LAPEDPIFPI IGSLFLRELI KEFGTDHIYG ADTFNEMQPP SSEPSYLAAA TTAVYEAMTA VDTEAVWLLQ GWLFQHQPQF WGPAQIRAVL GAVPRGRLLV LDLFAESQPV YTRTASFQGQ PFIWCMLHNF GGNHGLFGAL EAVNGGPEAA RLFPNSTMVG TGMAPEGISQ NEVVYSLMAE LGWRKDPVPD LAAWVTSFAA RRYGVSHPDA GAAWRLLLRS VYNCSGEACR GHNRSPLVRR PSLQMNTSIW YNRSDVFEAW RLLLTSAPSL ATSPAFRYDL LDLTRQAVQE LVSLYYEEAR SAYLSKELAS LLRAGGVLAY ELLPALDEVL ASDSRFLLGS WLEQARAAAV SEAEADFYEQ NSRYQLTLWG PEGNILDYAN KQLAGLVANY YTPRWRLFLE ALVDSVAQGI PFQQHQFDKN VFQLEQAFVL SKQRYPSQPR GDTVDLAKKI FLKYYPRWVA GSW |
| 预测分子量 | 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NAGLU重组蛋白的3篇参考文献概览：
1. **文献名称**: "Production and characterization of recombinant human NAGLU-IGF2 fusion protein for enzyme replacement therapy in Sanfilippo B syndrome"
**作者**: Wang RY et al.
**摘要**: 研究报道了一种新型NAGLU-IGF2融合蛋白的制备，通过结合胰岛素样生长因子2(IGF2)增强细胞摄取效率，在体外和小鼠模型中显著提高了酶活性并减少糖胺聚糖蓄积。
2. **文献名称**: "Stabilization of recombinant human NAGLU through formulation optimization for potential intrathecal delivery"
**作者**: Li C et al.
**摘要**: 探索了重组NAGLU的制剂稳定性，发现添加海藻糖和聚山梨酯80可显著提高酶的热稳定性与半衰期，为鞘内给药治疗中枢神经系统病变提供了可行性支持。
3. **文献名称**: "Efficacy of AAV-mediated gene therapy delivering recombinant NAGLU in a murine model of Sanfilippo B"
**作者**: Ellinwood NM et al.
**摘要**: 利用腺相关病毒(AAV)载体递送重组NAGLU基因至Sanfilippo B模型小鼠，结果显示长期酶活性恢复、神经病理学改善及行为障碍缓解，验证了基因治疗的潜力。
**Background of Recombinant NAGLU Protein**
Recombinant NAGLU (alpha-N-acetylglucosaminidase) is a lysosomal enzyme encoded by the *NAGLU* gene, essential for the stepwise degradation of heparan sulfate, a glycosaminoglycan (GAG). In lysosomes, NAGLU cleaves the terminal alpha-linked N-acetylglucosamine residues from heparan sulfate, enabling its complete breakdown. Mutations in *NAGLU* lead to a deficiency in this enzyme, resulting in the accumulation of undegraded heparan sulfate within lysosomes. This metabolic disruption causes Sanfilippo syndrome type B (mucopolysaccharidosis type IIIB), a rare, progressive neurodegenerative lysosomal storage disorder characterized by severe neurological decline, developmental delays, and early mortality.
Recombinant NAGLU is produced using biotechnological platforms, such as mammalian cell cultures (e.g., CHO or HEK293 cells) or bacterial systems, to express the human *NAGLU* gene. This engineered protein is designed for enzyme replacement therapy (ERT), a therapeutic strategy aimed at restoring functional NAGLU activity in patients. However, ERT for Sanfilippo B faces challenges, including the enzyme’s limited ability to cross the blood-brain barrier (BBB) and reach affected central nervous system tissues. Modifications, such as fusion with BBB-targeting peptides or encapsulation in nanoparticle carriers, are being explored to enhance delivery.
Beyond ERT, recombinant NAGLU serves as a critical tool for studying disease mechanisms, screening potential drugs, and optimizing gene therapy approaches. Its production and characterization also support biomarker development and preclinical testing. Ongoing research focuses on improving stability, bioavailability, and targeting efficiency to address unmet clinical needs in Sanfilippo syndrome and related lysosomal disorders.
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