Correction of a genetic defect in multipotent germline stem cells using a human artificial chromosome
by
Kazuki Y, Hoshiya H, Kai Y, Abe S, Takiguchi M, Osaki M, Kawazoe S, Katoh M, Kanatsu-Shinohara M,
Inoue K, Kajitani N, Yoshino T, Shirayoshi Y, Ogura A, Shinohara T, Barrett JC, Oshimura M.
Department of Biomedical Science,
Institute of Regenerative Medicine and Biofunction,
Graduate School of Medical Science,
Tottori University, Yonago, Tottori, Japan.
Gene Ther. 2008 Apr;15(8):617-24.

ABSTRACT

Human artificial chromosomes (HACs) have several advantages as gene therapy vectors, including stable episomal maintenance that avoids insertional mutations and the ability to carry large gene inserts including regulatory elements. Multipotent germline stem (mGS) cells have a great potential for gene therapy because they can be generated from an individual's testes, and when reintroduced can contribute to the specialized function of any tissue. As a proof of concept, we herein report the functional restoration of a genetic deficiency in mouse p53-/- mGS cells, using a HAC with a genomic human p53 gene introduced via microcell-mediated chromosome transfer. The p53 phenotypes of gene regulation and radiation sensitivity were complemented by introducing the p53-HAC and the cells differentiated into several different tissue types in vivo and in vitro. Therefore, the combination of using mGS cells with HACs provides a new tool for gene and cell therapies. The next step is to demonstrate functional restoration using animal models for future gene therapy.

HAC
Biohappiness
Eugenics talk
Reprogenetics
Heritable HACs
'Designer babies'
Private eugenics
Preimplantation genetic diagnosis
5-HTTPR polymorphism/depression
Francis Galton and contemporary eugenics
Gene therapy and performance enhancement
The commercialisation of pre-natal enhancement
Transhumanism (H+): toward a Brave New World?
Human artificial chromosome with dystrophin gene

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