Emerging Issues in AAV-Mediated In Vivo Gene Therapy

作者：Pasqualina Colella, Giuseppe Ronzitti, Federico Mingozzi · 发表于：Molecular Therapy — Methods & Clinical Development · 年份：2017 · DOI：10.1016/j.omtm.2017.11.007 · 被引用次数：885 · 研究领域：Virus-based gene therapy research、RNA Interference and Gene Delivery、CRISPR and Genetic Engineering

applications, and present key achievements and emerging issues in the field. We will use the liver as a model target tissue for gene transfer based on the large amount of data available from preclinical and clinical studies.