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Gene Therapy: Optimising DNA delivery to the nucleus

Johnson-Saliba, Melanie; Jans, David A

Description

Gene therapy, the expression in cells of genetic material that has therapeutic activity, holds great promise for the treatment of a number of human diseases. A gene delivery vehicle, or vector, that may be of viral or non-viral origin, is generally used to carry the genetic material. Viral vectors have been developed that exclude immunogenic genes while taking advantage of the genes responsible for proficient integration of the viral genome into that of the host. In this way, viral vectors...[Show more]

dc.contributor.authorJohnson-Saliba, Melanie
dc.contributor.authorJans, David A
dc.date.accessioned2015-12-13T23:26:35Z
dc.date.available2015-12-13T23:26:35Z
dc.identifier.issn1389-4501
dc.identifier.urihttp://hdl.handle.net/1885/92904
dc.description.abstractGene therapy, the expression in cells of genetic material that has therapeutic activity, holds great promise for the treatment of a number of human diseases. A gene delivery vehicle, or vector, that may be of viral or non-viral origin, is generally used to carry the genetic material. Viral vectors have been developed that exclude immunogenic genes while taking advantage of the genes responsible for proficient integration of the viral genome into that of the host. In this way, viral vectors improve the probability of long-term expression of the therapeutic gene, whereas non-viral vectors, that are not as efficient at introducing and maintaining foreign gene expression, have the advantage of being non-pathogenic and non-immunogenic. Although thousands of patients have been involved in clinical trials for gene therapy, using hundreds of different protocols, true success has been limited. A major limitation of gene therapy approaches, especially when non-viral vectors are used, is the poor efficiency of DNA delivery to the nucleus; a crucial step to ensure ultimate expression of the therapeutic gene product. Here we review existing gene delivery approaches and, in particular, explore the possibility of enhancing non-viral gene delivery to the nucleus by incorporating specific nuclear targeting sequences in vectors, using a range of different strategies.
dc.publisherBentham Science Publishers Ltd
dc.sourceCurrent Drug Targets
dc.subjectKeywords: DNA; drug vehicle; liposome; virus vector; Adeno associated virus; Adenovirus; article; cancer; cell nucleus; clinical protocol; controlled study; cystic fibrosis; DNA vector; electroporation; gene activity; gene expression; gene sequence; gene targeting;
dc.titleGene Therapy: Optimising DNA delivery to the nucleus
dc.typeJournal article
local.description.notesImported from ARIES
local.description.refereedYes
local.identifier.citationvolume2
dc.date.issued2001
local.identifier.absfor100401 - Gene and Molecular Therapy
local.identifier.ariespublicationMigratedxPub26155
local.type.statusPublished Version
local.contributor.affiliationJohnson-Saliba, Melanie, College of Medicine, Biology and Environment, ANU
local.contributor.affiliationJans, David A, College of Medicine, Biology and Environment, ANU
local.bibliographicCitation.startpage371
local.bibliographicCitation.lastpage399
local.identifier.doi10.2174/1389450013348245
dc.date.updated2015-12-12T09:47:13Z
local.identifier.scopusID2-s2.0-0035192233
CollectionsANU Research Publications

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