title: Human artificial chromosomes for Duchenne muscular dystrophy and beyond: challenges and hopes.
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.
authors.affiliation: Department of Cell and Developmental Biology, University College London, 21 University Street, London, WC1E 6DE, UK, f.s.tedesco@ucl.ac.uk.
authors.forename: Francesco Saverio
authors.initials: FS
authors.lastname: Tedesco
mesh_terms: D022222:Chromosomes, Artificial, Human / Q000235:genetics*; D005818:Genetic Engineering / Q000379:methods*; D015316:Genetic Therapy / Q000379:methods* / Q000639:trends; D005822:Genetic Vectors / Q000235:genetics*; D006801:Humans; D057026:Induced Pluripotent Stem Cells / Q000378:metabolism*; D020388:Muscular Dystrophy, Duchenne / Q000235:genetics* / Q000628:therapy