Human artificial chromosome (HAC) vector with a conditional centromere for correction of genetic deficiencies in human cells.

ABSTRACT: Human artificial chromosome (HAC)-based vectors offer a promising system for delivery and expression of full-length human genes of any size. HACs avoid the limited cloning capacity, lack of copy number control, and insertional mutagenesis caused by integration into host chromosomes that plague viral vectors. We previously described a synthetic HAC that can be easily eliminated from cell populations by inactivation of its conditional kinetochore. Here, we demonstrate the utility of this HAC, which has a unique gene acceptor site, for delivery of full-length genes and correction of genetic deficiencies in human cells. A battery of functional tests was performed to demonstrate expression of NBS1 and VHL genes from the HAC at physiological levels. We also show that phenotypes arising from stab

SUBMITTER: Kim JH 

PROVIDER: S-EPMC3250132 | biostudies-literature | 2011 Dec

REPOSITORIES: biostudies-literature