干细胞递送疗法治疗脑部疾病。
Stem cell delivery of therapies for brain disorders.
机构信息
Graduate School of Biomedical Sciences, Texas, USA.
Graduate School of Biomedical Sciences, Texas, USA ; Department of Medicine - Division of Hematology/Oncology, New Jersey Medical School, Rutgers School of Biomedical Health Science, Newark, NJ 07103, USA.
出版信息
Clin Transl Med. 2014 Jul 19;3:24. doi: 10.1186/2001-1326-3-24. eCollection 2014.
The blood brain barrier (BBB) poses a problem to deliver drugs for brain malignancies and neurodegenerative disorders. Stem cells such as neural stem cells (NSCs) and mesenchymal stem cells (MSCs) can be used to delivery drugs or RNA to the brain. This use of methods to bypass the hurdles of delivering drugs across the BBB is particularly important for diseases with poor prognosis such as glioblastoma multiforme (GBM). Stem cell treatment to deliver drugs to neural tumors is currently in clinical trial. This method, albeit in the early phase, could be an advantage because stem cells can cross the BBB into the brain. MSCs are particularly interesting because to date, the experimental and clinical evidence showed 'no alarm signal' with regards to safety. Additionally, MSCs do not form tumors as other more primitive stem cells such as embryonic stem cells. More importantly, MSCs showed pathotropism by migrating to sites of tissue insult. Due to the ability of MSCs to be transplanted across allogeneic barrier, drug-engineered MSCs can be available as off-the-shelf cells for rapid transplantation. This review discusses the advantages and disadvantages of stem cells to deliver prodrugs, genes and RNA to treat neural disorders.
血脑屏障(BBB)给脑部恶性肿瘤和神经退行性疾病的药物递送带来了难题。干细胞,如神经干细胞(NSCs)和间充质干细胞(MSCs),可用于将药物或 RNA 递送到大脑。这种绕过 BBB 药物递送障碍的方法对于预后不良的疾病尤为重要,例如多形性胶质母细胞瘤(GBM)。干细胞治疗用于向神经肿瘤递送药物目前正在临床试验中。这种方法虽然处于早期阶段,但可能具有优势,因为干细胞可以穿过 BBB 进入大脑。MSCs 特别有趣,因为迄今为止,实验和临床证据表明在安全性方面没有“警报信号”。此外,MSCs 不像其他更原始的干细胞(如胚胎干细胞)那样形成肿瘤。更重要的是,MSCs 表现出向组织损伤部位迁移的组织趋向性。由于 MSCs 能够跨越同种异体障碍进行移植,因此经过药物修饰的 MSCs 可以作为现成的细胞进行快速移植。本文综述了干细胞将前药、基因和 RNA 递送到治疗神经疾病的优势和劣势。