The Adeno-Associated Virus - A Safe and Effective Vehicle For Liver-Specific Gene Therapy of Inherited and Non-Inherited Diseases.

The Adeno-Associated Virus - A Safe and Effective Vehicle For Liver-Specific Gene Therapy of Inherited and Non-Inherited Diseases. Curr Gene Ther. 2017 Mar 14;: Authors: Mak KY, Rajapaksha IG, Angus PW, Herath CB Abstract The first human adeno-associated virus (AAV) was originally discovered in 1960s as a contaminant of adenovirus stocks preparation and thus it had not been of medical interest. Throughout last three decades AAV has gained popularity to be used in gene therapy, mainly due to its replicative defectiveness and lack of pathogenicity in human. In addition, the ability to mediate a stable and long-term expression in both non-dividing and dividing cells with specific tissue tropism makes AAV as one of the most promising candidates for therapeutic gene transfer to treat many genetic as well as non-genetic disorders. Moreover, the use of AAV is not only restricted to over-expression of recombinant transgene and protein, but also includes short hairpin RNAs and microRNAs to knock down the expression of genes in targeted tissues. This review will be organized into four parts. Firstly, we will discuss the discovery and history of AAV, followed by detailed AAV biology such as virus genome and virion structure and life cycle of AAV. In the second part of the review, molecular mechanisms of AAV tissue transduction will be discussed extensively, including receptor recognition and cell binding, endosomal and nucleus entry, virus unco...
Source: Current Gene Therapy - Category: Genetics & Stem Cells Authors: Tags: Curr Gene Ther Source Type: research