Gene transfer in skeletal and cardiac muscle using recombinant adeno-associated virus.
Gene transfer in skeletal and cardiac muscle using recombinant adeno-associated virus.
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DOI：
10.1002/9780471729259.mc14d03s28
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发表时间：
2013
影响因子：
--
通讯作者：
Gao, Guangping
中科院分区：
文献类型：
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作者：
Gruntman, Alisha M;Bish, Lawrence T;Mueller, Christian;Sweeney, H Lee;Flotte, Terence R;Gao, Guangping
腺相关病毒(AAV)是一种DNA病毒，其基因组大小为4.7kb。它是依赖病毒组的一种自然复制缺陷的细小病毒。重组AAV(RAAV)是通过用目的基因以及启动子和多聚腺苷化序列取代病毒的rep和帽基因而形成的基因转移载体。在生产过程中，顺式病毒只需要病毒反向末端重复序列(ITR)进行复制和包装。ITR对于载体基因组的处理和转导过程中的持久性也是必要的和充分的。RAAV载体的组织趋向性由AAV衣壳决定。在本单元中，我们将讨论几种转导心肌和/或骨骼肌的rAAV的方法，包括：静脉注射、肌肉注射、离体肢输注、新生小鼠心包内注射和成年大鼠左室壁注射。
Adeno-associated virus (AAV) is a DNA virus with a small (~4.7kb) single-stranded genome. It is a naturally replication-defective parvovirus of the dependovirus group. Recombinant AAV (rAAV), for use as a gene transfer vector, is created by replacing the viral rep and cap genes with the transgene of interest along with promoter and polyadenylation sequences. Only the viral inverted terminal repeats (ITRs) are required in cis for replication and packaging during production. The ITRs are also necessary and sufficient for vector genome processing and persistence during transduction. The tissue tropism of the rAAV vector is determined by the AAV capsid. In this unit we will discuss several methods to deliver rAAV in order to transduce cardiac and/or skeletal muscle, including: intravenous delivery, intramuscular delivery, isolated limb infusion, intrapericardial injection in neonatal mice, and left ventricular wall injection in adult rats.