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Development of new, biodegradable synthetic polymers as non-viral gene delivery systems for the treatment of ARMD

机译:研发可生物降解的新型合成聚合物作为非病毒基因递送系统,用于治疗ARMD

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Bacause of the increasing need and interest to treat gene-defected diseases and the potential toxicity of viral vectors, the search for non-viral gene delivery systems based on synthetic polymers is gaining considerable significance. For this research, the development and evaluation of non-immunogenic, biodegradable and multifunctional poly-a-amino acids was investigated. Both their physico-chemical properties as well as the capacity to act as a vector for retinal transfection applications (such as age-related macular degeneration, ARMD) is examined.
机译:由于治疗基因缺陷型疾病和病毒载体的潜在毒性的需求和兴趣不断增长,寻找基于合成聚合物的非病毒基因递送系统具有重要意义。对于这项研究,研究和开发了非免疫原性,可生物降解和多功能的聚α-氨基酸。检验了它们的理化性质以及用作视网膜转染应用(如年龄相关性黄斑变性,ARMD)的载体的能力。

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