Issa Shaza S, Shaimardanova Alisa A, Valiullin Victor V, Rizvanov Albert A, Solovyeva Valeriya V
Institute of Fundamental Medicine and Biology, Kazan Federal University, Kazan, Russia.
Faculty of Biology, Saint Petersburg State University, Saint Petersburg, Russia.
Front Pharmacol. 2022 Mar 2;13:859516. doi: 10.3389/fphar.2022.859516. eCollection 2022.
Lysosomal storage diseases (LSDs) are a group of approximately 50 genetic disorders caused by mutations in genes coding enzymes that are involved in cell degradation and transferring lipids and other macromolecules. Accumulation of lipids and other macromolecules in lysosomes leads to the destruction of affected cells. Although the clinical manifestations of different LSDs vary greatly, more than half of LSDs have symptoms of central nervous system neurodegeneration, and within each disorder there is a considerable variation, ranging from severe, infantile-onset forms to attenuated adult-onset disease, sometimes with distinct clinical features. To date, treatment options for this group of diseases remain limited, which highlights the need for further development of innovative therapeutic approaches, that can not only improve the patients' quality of life, but also provide full recovery for them. In many LSDs stem cell-based therapy showed promising results in preclinical researches. This review discusses using mesenchymal stem cells for different LSDs therapy and other neurodegenerative diseases and their possible limitations.
溶酶体贮积症（LSDs）是一组约50种遗传性疾病，由编码参与细胞降解以及脂质和其他大分子转运的酶的基因突变引起。脂质和其他大分子在溶酶体中的积累会导致受影响细胞的破坏。尽管不同溶酶体贮积症的临床表现差异很大，但超过一半的溶酶体贮积症有中枢神经系统神经退行性变的症状，并且在每种疾病中都存在相当大的差异，从严重的婴儿期发病形式到成人期发病的轻症，有时还有明显的临床特征。迄今为止，这类疾病的治疗选择仍然有限，这凸显了进一步开发创新治疗方法的必要性，这些方法不仅可以提高患者的生活质量，还能使他们完全康复。在许多溶酶体贮积症中，基于干细胞的疗法在临床前研究中显示出有前景的结果。本综述讨论了使用间充质干细胞治疗不同的溶酶体贮积症和其他神经退行性疾病及其可能的局限性。
