Advances in Recombinant Adeno-Associated Viral Vectors for Gene Delivery

被引：25

作者：

Petrs-Silva, Hilda ^{[1
]}

Linden, Rafael ^{[1
]}

机构：

[1] Univ Fed Rio de Janeiro, Inst Biofis, Rio de Janeiro, Brazil

来源：

CURRENT GENE THERAPY | 2013年 / 13卷 / 05期

关键词：

Viral vectors; adeno-associated virus; directed evolution; selective mutagenesis; gene transfer; safety; efficacy; ADENO-ASSOCIATED VIRUS; SEVERE COMBINED IMMUNODEFICIENCY; AAV2 CAPSID GENE; DIRECTED EVOLUTION; IN-VIVO; TRANSDUCTION EFFICIENCY; SEROTYPE VECTORS; SKELETAL-MUSCLE; DNA-SYNTHESIS; MOUSE RETINA;

D O I：

10.2174/15665232113136660028

中图分类号：

Q3 [遗传学];

学科分类号：

071007 ; 090102 ;

摘要：

Recombinant adeno-associated viral vectors (rAAV) have now been used in several clinical trials to treat a variety of diseases, and are currently the preferred choice of many investigators in the field, due to both their low pathogenicity and immunogenicity compared with other viral vectors, as well as localized long-term gene expression, despite their limitations of DNA size packaging and speed of expression. Recently, a number of advances have led to new generations of rAAV vectors, with improved features. This review addresses the various strategies employed to such effect, namely exploring distinct serotype tropisms, the production of mosaic and chimeric capsids, the selection of vectors through directed evolution, the development of self-complementary vectors, the use of pharmacological adjuvants and the induction of specific capsid mutations. Such approaches are expected to help the establishment of rAAV-based clinical gene therapy in the near future.

引用

页码：335 / 345

页数：11

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