Treatment of lysosomal storage disorders: Focus on the neuronal ceroid-lipofuscinoses.

Pierret C; Morrison JA; Kirk MD

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Treatment of lysosomal storage disorders: Focus on the neuronal ceroid-lipofuscinoses.

机译：溶酶体贮积症的治疗：着重于神经元类固醇脂褐藻糖。

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Recent advances in our understanding of lysosomal storage disorders (LSDs) may lead to new therapies to treat the neuronal ceroid-lipofuscinoses (NCLs). In this review, enzyme replacement therapy, gene therapy, cell-mediated therapy and pharmaceutical treatments are considered across the LSDs and extended to therapies for the NCLs. It is likely that a combination of approaches will produce the most beneficial clinical outcome for treatment of pathologies displayed by the NCLs.

机译：我们对溶酶体贮积症（LSD）的了解的最新进展可能会导致新的疗法来治疗神经元类固醇-脂褐藻糖糖（NCL）。在这篇综述中，酶替代疗法，基因疗法，细胞介导疗法和药物疗法被认为贯穿了整个LSD，并扩展到了NCL的疗法中。多种方法的组合可能会产生最有益的临床结果，以治疗NCL所显示的病理。

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《Acta neurobiologiae experimentalis》 |2008年第3期|共14页
作者
Pierret C; Morrison JA; Kirk MD;
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正文语种 eng
中图分类人体生理学;
关键词
Therapeutic procedure; Lipofuscin; Ceroid; enzyme replacement therapy; Recent; 脂褐质; 蜡样脂;

机译：Therapeutic procedure;Lipofuscin;Ceroid;enzyme replacement therapy;Recent;脂褐质;蜡样脂;

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1. Treatment of lysosomal storage disorders: Focus on the neuronal ceroid-lipofuscinoses. [J] . Pierret C, Morrison JA, Kirk MD Acta neurobiologiae experimentalis . 2008,第3期

机译：溶酶体贮积症的治疗：着重于神经元类固醇脂褐藻糖。
2. Thematic review series: Recent advances in the treatment of lysosomal storage diseases: Gene therapy for the neurological manifestations in lysosomal storage disorders [J] . ChengS.H. Journal of Lipid Research . 2014,第9期

机译：专题回顾系列：溶酶体贮积病治疗的最新进展：溶酶体贮积病神经系统表现的基因治疗
3. Lysosomal storage disorder 4+1 multiplex assay for newborn screening using tandem mass spectrometry: Application to a small-scale population study for five lysosomal storage disorders [J] . OrsiniJ.J., MartinM.M., ShowersA.L., Clinica chimica acta: International journal of clinical chemistry and applied molecular biology . 2012,第15a16期

机译：用于串联筛查新生儿筛查的溶酶体贮积病4 + 1多重测定：在五个溶酶体贮积病的小规模人群研究中的应用
4. APPROACH TO LYSOSOMAL STORAGE DISEASES IN THE DOG: THE NEURONAL CEROID LIPOFUSCINOSES [C] . Dennis OBrien American College of Veterinary Internal Medicine Forum . 2010

机译：狗溶酶体储存疾病的方法：神经元曲线脂肪属
5. Potential therapeutics for lysosomal storage disorders: Chemical biology dissection of cholesterol homeostasis mechanisms in mammalian cells. [D] . Rosenbaum, Anton I. 2011

机译：溶酶体贮积病的潜在疗法：哺乳动物细胞中胆固醇稳态机制的化学生物学解剖。
6. Thematic Review Series: Recent Advances in the Treatment of Lysosomal Storage Diseases: Lysosomal exocytosis and lipid storage disorders [O] . Mohammad Ali Samie, Haoxing Xu 2014

机译：专题回顾系列：溶酶体贮积病治疗的最新进展：溶酶体胞吐和脂质贮积病
7. Clinical Utility of Neuronal Cells Directly Converted from Fibroblasts of Patients for Neuropsychiatric Disorders: Studies of Lysosomal Storage Diseases and Channelopathy [O] . S. Kano, M. Yuan, R.A. Cardarelli, 2015

机译：从患者患者的成纤维细胞直接转化的神经元细胞的临床效用：溶酶体储存疾病和通道的研究

Treatment of lysosomal storage disorders: Focus on the neuronal ceroid-lipofuscinoses.

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