干细胞治疗神经退行性疾病

S. An, K. Sugaya
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引用次数: 3

摘要

神经退行性疾病是与年龄有关的破坏性疾病,严重影响患者和护理人员,并极大地增加了国家的经济负担。尽管在实验室和临床进行了数十年的努力,但专门为患者设计的有效治疗方法仍然遥不可及。干细胞治疗虽然存在一些挑战,包括成体干细胞的有限分化潜力、使用胚胎和胎儿干细胞的伦理问题、细胞移植后的肿瘤形成等,但为治疗几种神经退行性疾病提供了巨大的潜力。另一方面,目前市场上的药物主要是缓解症状,而不是治疗疾病本身。药物通过血脑屏障的效率、稳定性、疗效和这些药物对患者的副作用是药理学方法必须克服的一些障碍。在分子水平上详细了解这些复杂的疾病,然后正确结合专门定制的干细胞治疗和/或有效的药物,例如MS-818,用于增加内源性干细胞群,这可能是未来几年诊断后时间所至的患者的最佳行动方案。
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Stem Cell Approaches for Treatment of Neurodegenerative Diseases
Neurodegenerative diseases are devastating age-related disorders severely affecting the patient, caregivers, and enormously increasing the financial burden of the nation. Despite decades of hard work both in laboratory and clinic, the effective treatment specifically designed for a patient is still far from reach. Stem cell therapy, though with several challenges including limited differentiation potential of adult stem cells, ethical issues with using embryonic and fetal stem cells, tumor formation upon transplantation of cells, etc., offers enormous potential for treatment of several neurodegenerative diseases. Pharmacological drugs currently available in the market on the other hand are mainly for alleviating the symptoms and not for treating the disease per se. The efficiency of drug delivery across the bloodbrain barrier, stability, efficacy, and side effects these drugs show on patients are some of the hurdles pharmacological approach has to overcome. A detailed understanding of these complicated diseases at molecular level followed by the right combination of specifically tailored stem cell therapy and/or effective drugs e.g. MS-818 used to increase the endogenous stem cell population might be the best course of action in coming years for patients with little time left after their diagnosis.
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