Stem cell delivery of therapies for brain disorders.
Stem cell delivery of therapies for brain disorders.
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DOI：
10.1186/2001-1326-3-24
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发表时间：
2014
影响因子：
10.6
通讯作者：
Rameshwar P
中科院分区：
文献类型：
--
作者：
Aleynik A;Gernavage KM;Mourad YSh;Sherman LS;Liu K;Gubenko YA;Rameshwar P
血脑屏障(BBB)给治疗脑恶性肿瘤和神经退行性疾病的药物输送带来了问题。神经干细胞(NSCs)和间充质干细胞(MSCs)等干细胞可用于将药物或RNA输送到大脑。这种绕过血脑屏障给药障碍的方法对于预后较差的疾病特别重要，如多形性胶质母细胞瘤(GBM)。干细胞疗法将药物输送到神经肿瘤目前正在进行临床试验。这种方法虽然处于早期阶段，但可能是一种优势，因为干细胞可以穿过血脑屏障进入大脑。MSCs之所以特别有趣，是因为到目前为止，实验和临床证据表明，在安全性方面没有任何警报信号。此外，MSCs不会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通过迁移到组织损伤部位表现出致病性。由于MSCs可以跨同种异体屏障移植，药物工程MSCs可以作为现成的细胞用于快速移植。本文就干细胞运送前体药物、基因和RNA治疗神经疾病的优缺点作一综述。
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.