생화학분자생물학회

	Name: Dai-Wu Seol Email: seold@cau.ac.kr
	2010-present	Professor, College of Pharmacy, CHUNG-ANG University
	2007-2010	Associate Professor, Department of Life Sciences, KYUNGWON University (Currently, GACHEON University)
	2007-2015	Adjunct Professor, University of Pittsburgh School of Medicine, USA
	2000-2007	Assistant Professor, University of Pittsburgh School of Medicine, USA
	1998-2000	Research Associate, University of Pittsburgh School of Medicine, USA
	1992-1998	Ph.D., Department of Pathology, University of Pittsburgh School of Medicine, USA

Helper virus-free gutless adenovirus (HF-GLAd): a new platform for gene therapy

Gene therapy is emerging as a treatment option for inherited genetic diseases. The success of this treatment approach greatly depends upon gene delivery vectors. Researchers have attempted to harness the potential of viral vectors for gene therapy applications over many decades. Among the viral vectors available, gutless adenovirus (GLAd) has been recognized as one of the most promising vectors for in vivo gene delivery. GLAd is constructed by deleting all the viral genes from an adenovirus. Owing to this structural feature, the production of GLAd requires a helper that supplies viral proteins in trans. Conventionally, the helper is an adenovirus. Although the helper adenovirus efficiently provides helper functions, it remains as an unavoidable contaminant and also generates replication-competent adenovirus (RCA) during the production of GLAd. These two undesirable contaminants have raised safety concerns and hindered the clinical applications of GLAd. Recently, we developed helper virus-free gutless adenovirus (HF-GLAd), a new version of GLAd, which is produced by a helper plasmid instead of a helper adenovirus. Utilization of this helper plasmid eliminated the helper adenovirus and RCA contamination in the production of GLAd. HF-GLAd, devoid of helper adenovirus and RCA contaminants, will facilitate its clinical applications. In this review, we discuss the characteristics of adenoviruses, the evolution and production of adenoviral vectors, and the unique features of HF-GLAd as a new platform for gene therapy. Furthermore, we highlight the potential applications of HF-GLAd as a gene delivery vector for the treatment of various inherited genetic diseases.

https://pubmed.ncbi.nlm.nih.gov/32958121/
BMB Rep 2020 Sep 22;5157. Online ahead of print