Department of Pharmacology, University of Texas Health Science Center at San Antonio, San Antonio, Texas, USA.
Center for Biomedical Neuroscience, University of Texas Health Science Center at San Antonio, San Antonio, Texas, USA.
Stem Cells Dev. 2020 Sep 1;29(17):1118-1130. doi: 10.1089/scd.2019.0265. Epub 2020 Mar 5.
Treatment options for neurodevelopmental disorders such as schizophrenia and autism are currently limited. Antipsychotics used to treat schizophrenia are not effective for all patients, do not target all symptoms of the disease, and have serious adverse side effects. There are currently no FDA-approved drugs to treat the core symptoms of autism. In an effort to develop new and more effective treatment strategies, stem cell technologies have been used to reprogram adult somatic cells into induced pluripotent stem cells, which can be differentiated into neuronal cells and even three-dimensional brain organoids. This new technology has the potential to elucidate the complex mechanisms that underlie neurodevelopmental disorders, offer more relevant platforms for drug discovery and personalized medicine, and may even be used to treat the disease.
目前,针对精神分裂症和自闭症等神经发育障碍的治疗选择有限。用于治疗精神分裂症的抗精神病药物并非对所有患者都有效,无法针对该疾病的所有症状,并且具有严重的不良反应。目前,尚无 FDA 批准的药物可治疗自闭症的核心症状。为了开发新的、更有效的治疗策略,干细胞技术已被用于将成人成体细胞重编程为诱导多能干细胞,这些细胞可分化为神经元细胞,甚至三维脑类器官。这项新技术有可能阐明神经发育障碍的复杂机制,为药物发现和个性化医疗提供更相关的平台,甚至可能用于治疗该疾病。