2009PubMedRequires access

[Progress of human gene therapy].

Yasufumi Kaneda

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Abstract

Much attention has been paid to gene therapy since 1990's. No remarkable success has been achieved and several serious adverse effects such as leukemia in X-SCID gene therapy have been reported. Nevertheless, trials to treat hereditary disease, cancers and cardiovascular disease using therapeutic gene-loaded vectors are continuously being performed. Recently, some gene drugs such as p53-loaded adenovirus vector, oncolytic adenovirus and HGF plasmid DNA are produced and remain to be commercially available. Stem cell-based gene therapy will be main focus in hereditary disease gene therapy. In cancer gene therapy, regulation of anti-tumor immunity and improvement of gene-modified oncolytic virus will be more fully investigated.

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What this paper is about

Much attention has been paid to gene therapy since 1990's. No remarkable success has been achieved and several serious adverse effects such as leukemia in X-SCID gene therapy have been reported. Nevertheless, trials to treat hereditary disease, cancers and cardiovascular disease using therapeutic gene-loaded vectors are continuously being performed. Recently, some gene drugs such as p53-loaded adenovirus vector, oncolytic adenovirus and HGF plasmid DNA are produced and remain to be commercially available. Stem cell-based gene therapy will be main focus in hereditary disease gene therapy. In cancer gene therapy, regulation of anti-tumor immunity and improvement of gene-modified oncolytic virus will be more fully investigated.

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Available abstract

Much attention has been paid to gene therapy since 1990's. No remarkable success has been achieved and several serious adverse effects such as leukemia in X-SCID gene therapy have been reported. Nevertheless, trials to treat hereditary disease, cancers and cardiovascular disease using therapeutic gene-loaded vectors are continuously being performed. Recently, some gene drugs such as p53-loaded adenovirus vector, oncolytic adenovirus and HGF plasmid DNA are produced and remain to be commercially available. Stem cell-based gene therapy will be main focus in hereditary disease gene therapy. In cancer gene therapy, regulation of anti-tumor immunity and improvement of gene-modified oncolytic virus will be more fully investigated.

Key concepts: Genetic enhancement, Oncolytic virus, Oncolytic adenovirus, Medicine, Disease, Gene, Viral vector, Cancer research

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