Strategies for engineering human chromosomes with therapeutic potential.
Human engineered chromosomes (HECs) have several potential advantages over currently used vectors for gene therapy applications. Firstly, there is no upper size limit to DNA that can be cloned in these vectors. Secondly, their extrachromosomal nature ensures that introduced genes are neither disruptive to, nor affected by, the genome of the host cell. Finally, being solely human in origin, HEC vectors should not evoke adverse host immunogenic responses. Recent advances have produced a variety of HECs via several different approaches. This review focuses on the current methodologies for making HEC vectors, the advantages and problems associated with each strategy, and discusses the outlook for HEC vectors as ex vivo therapeutic agents.
Saffery
Richard
R
Murdoch Childrens Research Institute, Royal Childrens Hospital, Flemington Road, Parkville 3052, Australia. saffery@cryptic.rch.unimelb.edu.au
Choo
K H Andy
KH
D022222:Chromosomes, Artificial, Human; D053938:DNA Fragmentation; D004276:DNA, Satellite; D015316:Genetic Therapy / Q000379:methods* / Q000639:trends; D005822:Genetic Vectors; D006801:Humans
D004276:DNA, Satellite