Toward gene and cell therapy using human artificial chromosome]

Human artificial chromosomes (HACs) exhibit several potential characteristics desired for an ideal gene delivery vector, including stable episomal maintenance and the capacity to carry large genomic loci with their regulatory elements, thus allowing the physiological regulation of the introduced gene. Stem cells, including hematopoietic stem cells, bone marrow-derived MSCs and iPS cells, can potentially avoid immune rejection due to their being obtained from the patient's own tissues. Recently, we succeeded the complete correction of a genetic deficiency in iPS cells derived from a human Duchenne muscular dystrophy patient using the HAC technology. Therefore, the combination of patient specific stem cells and HAC containing defective genes potentially represents a powerful tool for gene and cell therapies.