caa a22 4500 606190317 CHVBK 20210128100854.0 cr unu---uuuuu 210128e20150201xx s 000 0 eng 10.1007/s10577-014-9460-6 doi (NATIONALLICENCE)springer-10.1007/s10577-014-9460-6 Tedesco Francesco Department of Cell and Developmental Biology, University College London, 21 University Street, WC1E 6DE, London, UK aut Human artificial chromosomes for Duchenne muscular dystrophy and beyond: challenges and hopes [Elektronische Daten] [Francesco Tedesco] 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. Springer Science+Business Media Dordrecht, 2014 Human Artificial Chromosome nationallicence Muscular dystrophy nationallicence Stem cells nationallicence Gene therapy nationallicence Cell therapy nationallicence AAV : Adeno-associated viral (vectors) nationallicence cDNA : Complementary DNA nationallicence CHO : Chinese hamster ovary (cells) nationallicence DMD : Duchenne muscular dystrophy nationallicence DT40 : Chicken B cell line (lymphoblasts) nationallicence DYS-HAC : Dystrophin-HAC nationallicence GFP : Green fluorescent protein nationallicence HAC : Human artificial chromosome nationallicence iPS : Induced pluripotent stem (cells) nationallicence MMCT : Microcell-mediated chromosome transfer nationallicence PEG : Polyethylene glycol nationallicence Chromosome Research Springer Netherlands 23/1(2015-02-01), 135-141 0967-3849 23:1<135 2015 23 10577 https://doi.org/10.1007/s10577-014-9460-6 text/html Onlinezugriff via DOI BK010053 XK010053 XK010000 Metadata rights reserved Springer special CC-BY-NC licence nationallicence 1 research-article jats NATIONALLICENCE NATIONALLICENCE NL-springer NATIONALLICENCE 856 40 https://doi.org/10.1007/s10577-014-9460-6 text/html Onlinezugriff via DOI NATIONALLICENCE 100 1- Tedesco Francesco Department of Cell and Developmental Biology, University College London, 21 University Street, WC1E 6DE, London, UK aut NATIONALLICENCE 773 0- Chromosome Research Springer Netherlands 23/1(2015-02-01), 135-141 0967-3849 23:1<135 2015 23 10577