Stepankova Katerina, Jendelova Pavla, Machova Urdzikova Lucia
Institute of Experimental Medicine, Czech Academy of Sciences, Vídeňská 1083, 14200 Prague, Czech Republic.
Department of Neuroscience, Second Faculty of Medicine, Charles University, 15006 Prague, Czech Republic.
Biomedicines. 2021 May 28;9(6):613. doi: 10.3390/biomedicines9060613.
The spinal cord injury (SCI) is a medical and life-disrupting condition with devastating consequences for the physical, social, and professional welfare of patients, and there is no adequate treatment for it. At the same time, gene therapy has been studied as a promising approach for the treatment of neurological and neurodegenerative disorders by delivering remedial genes to the central nervous system (CNS), of which the spinal cord is a part. For gene therapy, multiple vectors have been introduced, including integrating lentiviral vectors and non-integrating adeno-associated virus (AAV) vectors. AAV vectors are a promising system for transgene delivery into the CNS due to their safety profile as well as long-term gene expression. Gene therapy mediated by AAV vectors shows potential for treating SCI by delivering certain genetic information to specific cell types. This review has focused on a potential treatment of SCI by gene therapy using AAV vectors.
脊髓损伤（SCI）是一种会扰乱医疗和生活的病症，对患者的身体、社交和职业福祉造成毁灭性后果，且尚无足够的治疗方法。与此同时，基因治疗作为一种有前景的治疗神经和神经退行性疾病的方法，已通过将治疗性基因传递到中枢神经系统（CNS）（脊髓是其中一部分）进行了研究。对于基因治疗，已引入多种载体，包括整合型慢病毒载体和非整合型腺相关病毒（AAV）载体。AAV载体因其安全性以及长期基因表达，是一种将转基因传递到中枢神经系统的有前景的系统。由AAV载体介导的基因治疗通过将特定遗传信息传递到特定细胞类型，显示出治疗脊髓损伤的潜力。本综述重点关注使用AAV载体通过基因治疗对脊髓损伤的潜在治疗方法。
