Long-term correction of glycogen storage disease type II with a hybrid Ad-AAV vector.
Long-term correction of glycogen storage disease type II with a hybrid Ad-AAV vector.
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使用混合 Ad-AAV 载体长期纠正 II 型糖原累积病。
DOI：
10.1016/s1525-0016(02)00055-2
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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,Bao-dong;Chen,Y-T;Bird,Andrew;Amalfitano,Andrea;Koeberl,DwightD
We administered an adenovirus-adeno-associated virus (Ad-AAV) vector encoding human acid α-glucosidase (hGAA) to acid α-glucosidase-knockout (GAA-KO) mice on day 3 of life by gastrocnemius injection. In contrast to previous results for muscle-targeted Ad vector in adult GAA-KO mice, the muscles of the hindlimb showed reduced glycogen content and persistent hGAA for as long as 6 months after neonatal Ad-AAV vector administration. Not only the injected gastrocnemius muscles, but also the hamstrings and quadriceps muscles produced therapeutic levels of hGAA as a result of widespread transduction with the Ad-AAV vector; moreover, hGAA activity was 50-fold elevated as compared to normal mice. Vector RNA was detected in the hindlimb muscles, the hearts, and the livers by northern blot analysis and/or by RT-PCR for as long as 6 months. The low levels of hGAA detected in the heart were attributable to transduction with the Ad-AAV vector, not to secretion of hGAA by the injected muscle and uptake by the heart. Finally, although an antibody response to hGAA was present, it did not prevent the correction of glycogen storage in the skeletal muscle of GAA-KO mice.
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影响因子：
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作者：
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10.1073/pnas.93.24.14082
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影响因子：
12.4
作者：
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作者：
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