Packaging of an AAV vector encoding human acid alpha-glucosidase for gene therapy in glycogen storage disease type II with a modified hybrid adenovirus-AAV vector.
Packaging of an AAV vector encoding human acid alpha-glucosidase for gene therapy in glycogen storage disease type II with a modified hybrid adenovirus-AAV vector.
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将编码人类酸性 α-葡萄糖苷酶的 AAV 载体与改良的杂合腺病毒-AAV 载体包装在一起，用于 II 型糖原贮积病的基因治疗。
DOI：
10.1016/s1525-0016(03)00022-4
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发表时间：
2003
期刊：
影响因子：
--
通讯作者：
Koeberl,DwightD
中科院分区：
文献类型：
--
作者：
Sun,Baodong;Chen,Y-T;Bird,Andrew;Xu,Fang;Hou,Yang-Xun;Amalfitano,Andrea;Koeberl,DwightD
We have developed an improved method for packaging adeno-associated virus (AAV) vectors with a replication-defective adenovirus-AAV (Ad-AAV) hybrid virus. The AAV vector encoding human acid α-glucosidase (hGAA) was cloned into an E1, polymerase/preterminal protein-deleted adenovirus, such that it is packaged as an Ad vector. Importantly, the Ad-AAV hybrid cannot replicate during AAV vector packaging in 293 cells, because of deletion of polymerase/preterminal protein. The residual Ad-AAV in the AAV vector stock was reduced to <1 infectious particle per 1010AAV vector particles. These modifications resulted in ∼30-fold increased packaging of the AAV vector for the hybrid Ad-AAV vector method as compared with standard transfection-only methods. Similarly improved packaging was demonstrated for pseudotyping the AAV vector as AAV6, and for AAV vector packaging with a second Ad-AAV vector encoding canine glucose-6-phosphatase. Liver-targeted delivery of either the Ad-AAV hybrid or AAV vector particles in acid α-glucosidase-knockout (GAA-KO) mice revealed secretion of hGAA with the Ad-AAV vector, and sustained secretion of hGAA with an AAV vector in hGAA-tolerant GAA-KO mice. Further development of hybrid Ad-AAV vectors could offer distinct advantages for gene therapy in glycogen storage diseases.
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影响因子：
8.3
作者：
MACCARTHY, EP;YAMASHITA, W;OOI, BS
通讯作者：
OOI, BS
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DOI：
--
发表时间：
1988
期刊：
影响因子：
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影响因子：
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DOI：
--
发表时间：
1988
期刊：
Proceedings of the Society for Experimental Biology and Medicine. Society for Experimental Biology and Medicine
影响因子：
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作者：
E. Young;R. Bukoski;D. McCarron
通讯作者：
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影响因子：
8.3
作者：
T. Oshima;E. Young;R. Bukoski;D. McCarron
通讯作者：
D. McCarron