A highly stable and nonintegrated human artificial chromosome (HAC) containing the 2.4 Mb entire human dystrophin gene.

@article{Hoshiya2009AHS,
  title={A highly stable and nonintegrated human artificial chromosome (HAC) containing the 2.4 Mb entire human dystrophin gene.},
  author={Hidetoshi Hoshiya and Yasuhiro Kazuki and Satoshi Abe and Masato Takiguchi and Naoyo Kajitani and Yoshinori Watanabe and Toko Yoshino and Yasuaki Shirayoshi and Katsumi Higaki and Graziella Messina and Giulio Cossu and Mitsuo Oshimura},
  journal={Molecular therapy : the journal of the American Society of Gene Therapy},
  year={2009},
  volume={17 2},
  pages={309-17}
}
Episomal vector with the capacity to deliver a large gene containing all the critical regulatory elements is ideal for gene therapy. Human artificial chromosomes (HACs) have the capacity to deliver an extremely large genetic region to host cells without integration into the host genome, thus preventing possible insertional mutagenesis and genomic instability. Duchenne muscular dystrophy (DMD) is caused by mutation in the extremely large dystrophin gene (2.4 Mb). We herein report the development… CONTINUE READING
Related Discussions
This paper has been referenced on Twitter 2 times. VIEW TWEETS

Citations

Publications citing this paper.
Showing 1-10 of 50 extracted citations

References

Publications referenced by this paper.
Showing 1-10 of 42 references

Similar Papers

Loading similar papers…