Emerging Issues in AAV-Mediated In Vivo Gene Therapy

Authors: Pasqualina Colella, Giuseppe Ronzitti, Federico Mingozzi - Molecular Therapy — Methods & Clinical Development 2017 cited by 864

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.