【摘 要】
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Mice with chimeric human-murine livers may be a better predictor (compared to any other animal model) of rAAV vector efficacy in humans This animal model ca
【出 处】
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The 3rd Canton Nucleic Acids Forum(第三届广州核酸国际论坛)
论文部分内容阅读
Mice with chimeric human-murine livers may be a better predictor (compared to any other animal model) of rAAV vector efficacy in humans This animal model can be used in combination with multi-species AAV capsid libraries to select for rAAV vectors with novel transduction properties A new rAAV vector capable of site-specific integration overcomes some of the limitations of canonical rAAV-based gene transfer and has been used to treat animal models of human disease These new vector strategies can be used to express therapeutic proteins or short-hairpin non-coding RNAs (e.g.Transcriptional RNAi).
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