Thomas Steininger1,2,3, Veronika Öttl1,4, Linda E Franken5

  • 1Roche Pharma Research and Early Development, Therapeutic Modalities, Roche Innovation Center Munich, Roche Diagnostics GmbH, Nonnenwald 2, 82377 Penzberg, Germany.

概括

工程设计的AAV Rep蛋白质增强了基因治疗载体的产生. 针对Rep变种的定向进化显著提高了病毒基因组包装效率,提高了总体载体产量.