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Evaluation of the Efficacy And Toxicity of RNAs Targeting HIV-1 Production for Use in Gene or Drug Therapy
Published on: September 5, 2016
Clinical Evaluations of Toxicity and Efficacy of Nanoparticle-Mediated Gene Therapy
Xiao Liang1, Li Liu2, Yu-Quan Wei1,2
11 Department of Gynecology and Obstetrics, Development and Related Diseases of Women and Children, Key Laboratory of Sichuan Province, Key Laboratory of Birth Defects and Related Diseases of Women and Children, Ministry of Education, West China Second Hospital, and State Key Laboratory of Biotherapy, West China Hospital, Sichuan University, Chengdu, China .
Abstract:
Considerable efforts have been devoted to develop safe and efficient gene therapies for life-threatening or inherited diseases. The choice of gene delivery vehicle plays key roles in enhancing the therapeutic effect of nucleic acid cargo. To date, gene therapy approaches involving both viral vectors and nonviral vectors have been evaluated in clinical trials. With improvements in material science and nanotechnologies, positively charged nanoparticles have emerged as potential gene delivery vehicles. In this review, we highlight clinical trials that examined cationic nanocarrier-mediated gene therapy as well as discuss both the toxicity and efficacy of nanocarrier-based therapeutics.
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