잠시만 기다려 주세요. 로딩중입니다.

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

BMB Reports 2020년 53권 11호 p.565 ~ 575
Liu Jida, 설대우,
소속 상세정보
 ( Liu Jida ) - Chung-Ang University College of Pharmacy
설대우 ( Seol Dai-Wu ) - Chung-Ang University College of Pharmacy

Abstract


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 replicationcompetent 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.

키워드

Adenovirus; Gene therapy; Genetic disease; GLAd; Gutless adenovirus; Helper plasmid; Helper virus; HF-GLAd; RCA

원문 및 링크아웃 정보

등재저널 정보