996. Application of a Novel Human Artificial Chromosome (HAC) Vector to Gene Therapy Aimed at Erythropoietin (EPO) Replacement

In the conventional virus and non-viral vectors for gene therapy, it comes up with crucial issues to be solved; 1) The introduced vector might develop lesions on the host chromosome and the fears for safety such as the malignant transformation are mentioned, 2) The copy number of the introduced gene is not regulated, 3) Sufficient expression in the host cell is not sustained for long term, 4) The introduced gene is inactivated or suffers the effects of controlled sequence on the host chromosome.