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Human artificial chromosomes for Duchenne muscular dystrophy and beyond: challenges and hopes.

Tedesco, FS; (2015) Human artificial chromosomes for Duchenne muscular dystrophy and beyond: challenges and hopes. Chromosome Res , 23 (1) 135 - 141. 10.1007/s10577-014-9460-6. Green open access

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Abstract

Safe and efficacious vectors able to carry large or several transgenes are of key importance for gene therapy. Human artificial chromosomes can fulfil this essential requirement; moreover, they do not integrate into the host genome. However, drawbacks such as the low efficiency of chromosome transfer and their relatively complex engineering still limit their widespread use. In this article, I summarise the key steps that brought human artificial chromosomes into preclinical research for Duchenne muscular dystrophy, an X-linked, monogenic disorder. I will also review possible future pre-clinical and clinical perspectives for this technology.

Type: Article
Title: Human artificial chromosomes for Duchenne muscular dystrophy and beyond: challenges and hopes.
Location: Netherlands
Open access status: An open access version is available from UCL Discovery
DOI: 10.1007/s10577-014-9460-6
Publisher version: http://dx.doi.org/10.1007/s10577-014-9460-6
Language: English
Additional information: Metadata improved. File saved to S-Drive(July 2015). Can be deposited in Disco following a 12 month embargo period. (TF 9/7/15)
Keywords: Human Artificial Chromosome . Muscular dystrophy. Stemcells . Gene therapy. Cell therapy.
UCL classification: UCL > Provost and Vice Provost Offices
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Life Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Life Sciences > Div of Biosciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Life Sciences > Div of Biosciences > Cell and Developmental Biology
URI: http://discovery.ucl.ac.uk/id/eprint/1461051
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