Immune Response Mechanisms against AAV Vectors in Animal Models.
作者： Ashley T Martino , David M Markusic , None
DOI: 10.1016/J.OMTM.2019.12.008
关键词: Immunity 、 Transduction (genetics) 、 Acquired immune system 、 Computational biology 、 Biology 、 Immune system 、 Genetic enhancement 、 Transgene 、 Innate immune system 、 Adeno-associated virus
摘要: Early preclinical studies in rodents and other species did not reveal that vector or transgene immunity would present a significant hurdle for sustained gene expression. While there was early evidence of mild immune responses to adeno-associated virus (AAV) studies, it generally believed these were too weak transient negatively impact transduction. However, translation the cumulative success treating hemophilia B dogs with an AAV2-F9 human as successful. Despite progress recent clinical trials hemophilia, new immunotoxicities AAV are emerging humans require better animal models assess overcome responses. The designed address complications have provided critical information how dose, capsid processing, genome, difference serotypes, variations delivery route can develop approaches overcoming pre-existing immunity. Additionally, comprehensive dissection innate, adaptive, regulatory vectors has framework be utilized development immunomodulatory therapies bypass developing strategic toward engineering stealth circumvent
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