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.
![[thumbnail of Tedesco.1461051__Chrom_Res__2015._accepted.pdf]](https://discovery-pp.ucl.ac.uk/style/images/fileicons/application_pdf.png) |
PDF
Tedesco.1461051__Chrom_Res__2015._accepted.pdf Download (289kB) |
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 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: | https://discovery-pp.ucl.ac.uk/id/eprint/1461051 |
Archive Staff Only
 |
View Item |
