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SITE-SPECIFIC INTEGRATING RECOMBINANT AAV VECTORS FOR GENE THERAPY AND IMPROVED PRODUCTION METHODS
SITE-SPECIFIC INTEGRATING RECOMBINANT AAV VECTORS FOR GENE THERAPY AND IMPROVED PRODUCTION METHODS
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机译:用于基因治疗的特定位点整合重组AAV矢量载体和改进的生产方法
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摘要
Provided herein are methods of site-specific integration of a heterologous sequence into a host genome (e.g., by administering a recombinant adeno-associated virus (rAAV) to a host cell in the presence of a Rep protein), and methods for treating diseases and disorders by delivering an rAAV that comprises a nucleic acid vector comprising a Rep protein. Also provided herein are methods and compositions for producing rAAV particles with improved titer and transduction efficiencies.
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