选择适当的非临床动物模型,以确保新型 AAV 基因疗法可转化为临床疗法。

IF 4.6 3区 医学 Q1 BIOCHEMISTRY & MOLECULAR BIOLOGY
Mark Singh, Andrew Brooks, Parto Toofan, Keith McLuckie
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引用次数: 0

摘要

基因治疗药物产品由一种用于调节或操作基因序列的重组核酸组成。新型基因疗法一次给药就有可能治愈疾病,并使患者长期持久受益。腺相关病毒载体具有轻微的免疫原性,能有效转导各种人体组织和细胞,而且基因组整合程度低,因此已成为体内传递治疗性转基因的首选病毒载体。将发现研究中产生的数据有效转化为临床研究的关键是选择合适的动物物种进行关键性非临床研究。本综述旨在支持选择合适的动物模型进行非临床研究,以推动新型腺相关病毒基因疗法的开发。
本文章由计算机程序翻译,如有差异,请以英文原文为准。

Selection of appropriate non-clinical animal models to ensure translatability of novel AAV-gene therapies to the clinic

Selection of appropriate non-clinical animal models to ensure translatability of novel AAV-gene therapies to the clinic

Selection of appropriate non-clinical animal models to ensure translatability of novel AAV-gene therapies to the clinic
Gene Therapy Medicinal Products consist of a recombinant nucleic acid intended for the modulation or manipulation of a genetic sequence. A single administration of a novel gene therapy has the potential to be curative, with a durable long-term benefit to patients. Adeno-associated viral vectors have become the viral vector of choice for in vivo delivery of therapeutic transgenes as they are mildly immunogenic, can effectively transduce a variety of human tissues and cells, and have low levels of genomic integration. Central to the effective translation of data generated in discovery studies to the clinic is the selection of appropriate animal species for pivotal non-clinical studies. This review aims to support the selection of appropriate animal models for non-clinical studies to advance the development of novel adeno-associated virus gene therapies.
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来源期刊
Gene Therapy
Gene Therapy 医学-生化与分子生物学
CiteScore
9.70
自引率
2.00%
发文量
67
审稿时长
4-8 weeks
期刊介绍: Gene Therapy covers both the research and clinical applications of novel therapeutic techniques based on a genetic component. Over the last few decades, significant advances in technologies ranging from identifying novel genetic targets that cause disease through to clinical studies, which show therapeutic benefit, have elevated this multidisciplinary field to the forefront of modern medicine.
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