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Current status of non-viral gene therapy for CNS disorders

机译:中枢神经系统疾病的非病毒基因治疗的现状

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Introduction: Viral and non-viral vectors have been used as methods of delivery in gene therapy for many CNS diseases. Currently, viral vectors such as adeno-associated viruses (AAV), retroviruses, lentiviruses, adenoviruses and herpes simplex viruses (HHV) are being used as successful vectors in gene therapy at clinical trial levels. However, many disadvantages have risen from their usage. Non-viral vectors like cationic polymers, cationic lipids, engineered polymers, nanoparticles, and naked DNA offer a much safer option and can therefore be explored for therapeutic purposes.
机译:简介:病毒和非病毒载体已被用作许多中枢神经系统疾病的基因治疗方法。当前,诸如腺伴随病毒(AAV),逆转录病毒,慢病毒,腺病毒和单纯疱疹病毒(HHV)之类的病毒载体已经在临床试验水平上用作基因治疗的成功载体。但是,使用它们带来了许多缺点。非病毒载体,如阳离子聚合物,阳离子脂质,工程聚合物,纳米颗粒和裸露的DNA提供了更为安全的选择,因此可以用于治疗目的。

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