Adipose Tissue: An Emerging Target for Adeno-associated Viral Vectors.
Adipose Tissue: An Emerging Target for Adeno-associated Viral Vectors.
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DOI：
10.1016/j.omtm.2020.09.009
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发表时间：
2020-12-11
期刊：
影响因子：
--
通讯作者：
Cao L
中科院分区：
文献类型：
--
作者：
Bates R;Huang W;Cao L
脂肪组织是人体最大的器官之一，在多种疾病的生理和病理中起着重要作用。然而，针对脂肪组织的腺相关病毒（AAV）的研究远远落后于对肝脏、大脑、心脏和肌肉的研究。尽管最初的报道表明表现不佳，但在过去的二十年中，aav介导的基因传递到脂肪组织的情况持续增加。迄今为止，AAV8和一种新的工程杂交血清型Rec2已被证明比其他血清型更有效地转导脂肪组织，并已应用于大多数体内研究。Rec2血清型显示，通过局部和全身给药，基因转移到棕色和白色脂肪的效率都很高。本文综述了增强向脂性和限制脱靶转基因表达的AAV载体的研究进展。我们讨论了为脂肪组织寻找和产生具有向性定制的新型血清型以及开发AAV载体系统以改善脂肪转基因表达的挑战和策略，用于基础研究和转化研究。与其他血清型相比，AAV8和一种新的杂交血清型Rec2在脂肪组织转导方面表现出更高的功效。本文综述了增强向脂性和限制脱靶转基因表达的AAV载体的研究进展。讨论了针对脂肪组织开发AAV载体系统的挑战和策略。
Adipose tissue is one of the largest organs, playing important roles in physiology and pathologies of multiple diseases. However, research related to adeno-associated virus (AAV) targeting adipose tissue has been left far behind studies carried out in the liver, brain, heart, and muscle. Despite initial reports indicating poor performance, AAV-mediated gene delivery to adipose tissue has continued to rise during the past two decades. AAV8 and a novel engineered hybrid serotype, Rec2, have been shown to transduce adipose tissue more efficiently than other serotypes so far tested and have been applied in most of the in vivo studies. The Rec2 serotype displays high efficacy of gene transfer to both brown and white fat via local and systemic administration. This review summarizes the advances in developing AAV vectors with enhanced adipose tropism and restricting off-target transgene expression. We discuss the challenges and strategies to search for and generate novel serotypes with tropism tailoring for adipose tissue and develop AAV vector systems to improve adipose transgene expression for basic research and translational studies. AAV8 and a novel hybrid serotype, Rec2, show higher efficacy in transducing adipose tissue than do other serotypes. This review summarizes the advances in developing AAV vectors with enhanced adipose tropism and restricting off-target transgene expression. The challenges and strategies to develop AAV vector systems tailoring for adipose tissue are discussed.
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