Abstract
Although homologous recombination has been used for gene correction in ES cells, genetic defects with an unknown mutation site or a genomic large deletion could not be restored by homologous recombination. Human artificial chromosomes (HACs) may solve these problems. HACs have several advantages that require for gene therapy vectors, including an efficient transfer to stem cells, a stable episomal maintenance and the capacity for large gene inserts. Although own embryonic stem (nt ES) cells can be made by the nuclear transfer technique, there are many ethical problems. On the other hand, mGS (multipotent germline stem) cells have ES cell-like multipotency and could be generated from neonatal mouse testis. Thus, mGS cells have greater advantages than ntES cells, because individual mGS cells can be obtained without ethical problem. Here we establish a paradigm for treatment of a genetic disorder by combining mGS cells with HAC vector containing a defected gene.
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