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“This monograph contains new and valuable information regarding relevant topics ranging from disease entities to vectors and engineered cells. The authors, all of them international experts, take into consideration the biomedical knowledge of these diseases and address the complexities of delivering long term genetic interventions. … The present volume is of an exceptional value for clinicians and scientists interested in introducing gene therapy into clinical use.” (Magdalena Moisei, Romanian Journal of Biochemistry, Vol. 47 (2), 2010)