Therapeutic Potential of Mesenchymal Stem Cells in Niemann-Pick Disease.

IF 2.4 4区生物学 Q3 BIOCHEMISTRY & MOLECULAR BIOLOGY

Molecular Biotechnology Pub Date : 2025-04-25 DOI:10.1007/s12033-025-01435-3

Keli Xu, Minlin Yang, Lihong Guan, Ciqing Yang, Liang Qiao, Yonghai Li, Juntang Lin, Xiaoying Li

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Abstract

Niemann-Pick disease (NPD) is a rare autosomal recessive neurodegenerative disease characterized by hepatosplenomegaly, neuropathy, and a significantly shortened lifespan. Lipid metabolism disorder is the main pathological feature of NPD. Currently, the exact pathogenesis of NPD remains unclear, and drug therapy is largely palliative, focusing on symptom management, but it has side effects. Mesenchymal stem cells (MSCs) possess several advantageous properties, including their differentiation potential, wide availability, low immunogenicity, and the ability to secrete regulatory factors, which have led to their extensive application in basic research targeting neurodegenerative diseases. Studies have demonstrated that transplantation of MSCs from different sources into animal models of NPD can delay the loss of Purkinje cells in the cerebellum, reduce lipid deposition, improve motor coordination, slow the rate of weight loss, and extend lifespan. This review explores the therapeutic potential of MSCs in the treatment of NPD, highlighting their emerging role in addressing this challenging condition.

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间充质干细胞治疗尼曼-匹克病的潜力。

尼曼-匹克病（NPD）是一种罕见的常染色体隐性神经退行性疾病，以肝脾肿大、神经病变和显著缩短的寿命为特征。脂质代谢紊乱是NPD的主要病理特征。目前，NPD的确切发病机制尚不清楚，药物治疗主要是姑息性的，侧重于症状管理，但它有副作用。间充质干细胞（MSCs）具有多种优势，包括其分化潜力、广泛可用性、低免疫原性和分泌调节因子的能力，这使得它们在针对神经退行性疾病的基础研究中得到广泛应用。研究表明，将不同来源的MSCs移植到NPD动物模型中，可以延缓小脑浦肯野细胞的丢失，减少脂质沉积，改善运动协调，减缓体重减轻的速度，延长寿命。这篇综述探讨了MSCs在治疗NPD中的治疗潜力，强调了它们在解决这一具有挑战性的疾病中的新作用。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Molecular Biotechnology 医学-生化与分子生物学

CiteScore

4.10

自引率

3.80%

发文量

165

审稿时长

6 months

期刊介绍： Molecular Biotechnology publishes original research papers on the application of molecular biology to both basic and applied research in the field of biotechnology. Particular areas of interest include the following: stability and expression of cloned gene products, cell transformation, gene cloning systems and the production of recombinant proteins, protein purification and analysis, transgenic species, developmental biology, mutation analysis, the applications of DNA fingerprinting, RNA interference, and PCR technology, microarray technology, proteomics, mass spectrometry, bioinformatics, plant molecular biology, microbial genetics, gene probes and the diagnosis of disease, pharmaceutical and health care products, therapeutic agents, vaccines, gene targeting, gene therapy, stem cell technology and tissue engineering, antisense technology, protein engineering and enzyme technology, monoclonal antibodies, glycobiology and glycomics, and agricultural biotechnology.