Please use this identifier to cite or link to this item: https://ahro.austin.org.au/austinjspui/handle/1/17553
Full metadata record
DC FieldValueLanguage
dc.contributor.authorMak, Kai Yan-
dc.contributor.authorRajapaksha, Indu G-
dc.contributor.authorAngus, Peter W-
dc.contributor.authorHerath, Chandana B-
dc.date.accessioned2018-05-02T01:04:24Z-
dc.date.available2018-05-02T01:04:24Z-
dc.date.issued2017-
dc.identifier.citationCurrent Gene Therapy 2017; 17(1): 4-16en_US
dc.identifier.urihttps://ahro.austin.org.au/austinjspui/handle/1/17553-
dc.description.abstractThe first human adeno-associated virus (AAV) was originally discovered in 1960s as a contaminant of adenovirus stock preparation and thus it had not been of medical interest. Throughout the 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, its ability to mediate stable and long-term expression in both non-dividing and dividing cells with specific tissue tropism makes AAV one of the most promising candidates for therapeutic gene transfer to treat many inherited as well as non-inherited disorders. Moreover, the use of AAV is not only restricted to overexpression of recombinant transgene, but also to over-express short hairpin RNA and microRNA to knockdown the expression of genes in targeted tissues. This review is organized into four parts. In the first part of the review, we discuss about the discovery and history of AAV, followed by detailed AAV biology such as virus genome, virus structure and its life cycle. In the second part of the review, the discussion is centred on the molecular mechanisms of AAV and tissue transduction, including receptor recognition and cell binding, endosomal entry, virus uncoating, nuclear entry and genome replication. Advantages and limitations of using AAV as a safe vehicle for gene delivery is also discussed. In the third part of the review, we discuss about the most commonly used AAV serotypes and variants isolated from human and non-human primates, focusing on their diverse tissue tropisms, transduction efficiency, immunological profiles and their applications in animal studies. Final part of the review focuses on the recent progress of in-vivo gene transfer using AAV for inherited and non-inherited diseases in both preclinical and clinical settings with a special emphasis on potential clinical applications of AAV in the field of liver disease.en_US
dc.language.isoeng-
dc.subjectAdeno-associated virusen_US
dc.subjectangiotensin converting enzyme 2en_US
dc.subjectgene therapyen_US
dc.subjectliveren_US
dc.subjectnon-inherited disordersen_US
dc.subjectrenin angiotensinsystemen_US
dc.subjectviral vectoren_US
dc.titleThe Adeno-associated Virus - A Safe and Promising Vehicle for Liverspecific Gene Therapy of Inherited and Non-inherited Disorders.en_US
dc.typeJournal Articleen_US
dc.identifier.journaltitleCurrent Gene Therapyen_US
dc.identifier.affiliationMedicine (University of Melbourne)en_US
dc.identifier.affiliationGastroenterology and Hepatologyen_US
dc.identifier.doi10.2174/1566523217666170314141931en_US
dc.type.contentTexten_US
dc.identifier.pubmedid28292253-
dc.type.austinJournal Article-
local.name.researcherAngus, Peter W
item.openairecristypehttp://purl.org/coar/resource_type/c_18cf-
item.cerifentitytypePublications-
item.fulltextNo Fulltext-
item.grantfulltextnone-
item.languageiso639-1en-
item.openairetypeJournal Article-
crisitem.author.deptVictorian Liver Transplant Unit-
crisitem.author.deptGastroenterology and Hepatology-
Appears in Collections:Journal articles
Show simple item record

Page view(s)

26
checked on Mar 28, 2024

Google ScholarTM

Check


Items in AHRO are protected by copyright, with all rights reserved, unless otherwise indicated.