Safety profile of recombinant adeno-associated viral vectors: focus on alipogene tiparvovec (Glybera)

被引：77

作者：

Salmon, Florence ^{[1
]}

Grosios, Konstantina ^{[1
]}

Petry, Harald ^{[1
]}

机构：

[1] UniQure, NL-1105 BA Amsterdam, Netherlands

来源：

EXPERT REVIEW OF CLINICAL PHARMACOLOGY | 2014年 / 7卷 / 01期

关键词：

adeno-associated viral vectors; alipogene tiparvovec; AAV1-LPLS447X; gene therapy; lipoprotein lipase; lipoprotein lipase deficiency; MUSCLE-SPECIFIC OVEREXPRESSION; HIGH-EFFICIENCY TRANSDUCTION; LONG-TERM CORRECTION; GENE-THERAPY; FACTOR-IX; LIPOPROTEIN-LIPASE; SKELETAL-MUSCLE; HEMOPHILIA-B; INTEGRATION SITES; VIRUS VECTORS;

D O I：

10.1586/17512433.2014.852065

中图分类号：

R9 [药学];

学科分类号：

1007 ;

摘要：

There has been great interest over the past two decades in developing gene therapies (GTs) to treat a variety of diseases; however, translating research findings into clinical treatments have proved to be a challenge. A major milestone in the development of GT has been achieved with the approval of alipogene tiparvovec (Glybera) in Europe for the treatment of familial lipoprotein lipase deficiency. At this important stage with the evolution of GT into the clinic, this review will examine the safety aspects GT with adeno-associated virus (AAV) vectors. The topics that will be covered include acute reactions, immunological reactions to the AAV capsid and expressed transgene, viral biodistribution and shedding, DNA integration and carcinogenicity. These safety aspects of GT will be discussed with a focus on alipogene tiparvovec, in addition to other AAV vector GT products currently in clinical development.

引用

页码：53 / 65

页数：13

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