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December 10, 2009

iPS cell technology used in gene therapy approach to Duchenne muscular dystrophy

December 9, 2009 - A new collaborative study between Mitsuo Oshimura and Yasuhiro Kazuki, et al. of Tottori University and CiRA scientists, led by CiRA director Shinya Yamanaka, has opened new avenues toward the treatment of a serious degenerative disease known as Duchenne muscular dystrophy (DMD). The team of scientists used a human artificial chromosome vector to introduce genes capable of correcting the genetic deficiency that underlies this disease into induced pluripotent stem (iPS) cells derived from mouse models of DMD and a human patient.

Researchers from CiRA contributed with technical guidance and review of the experimental results. This work was published in the journal Molecular Therapy.

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