Generation of gene therapy method for ataxia-telangiectasia using a human artificial chromosome

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To generate gene therapy vector for ataxia telangiectasia (AT), we constructed novel human artificial chromosome (HAC) containing ATM gene, which is mutated in ataxia telangiectasia (AT) patients. The ATM-HAC were transferred into ATM-deficient cell line derived from a AT patient. The ATM-deficient phenotypes of gene regulation and radiation sensitivity were complemented by introducing the ATM-HAC. Furthermore, the ATM-HAC was stably maintained in the cell lines. Thus, the ATM-HAC vector will be useful for gene therapy of AT.