A Transgenic Mouse Model for DNA/RNA Gene Therapy of Human β Thalassemia

Authors

  • Li Lu Main Laboratory, ZhongShan Ophthalmic Center of Sun Yan-sen University, GuangZhou and GuangDong Pharmaceutical University, GuangZhou
  • Bing Huang Main Laboratory, ZhongShan Ophthalmic Center of Sun Yan-sen University, GuangZhou
  • XiGu Chen Experimental Animal Center of Sun Yan-sen University, GuangZhou
  • XiangCai Wei Experimental Animal Center of Sun Yan-sen University, GuangZhou
  • GuoZhu Yang GuangDong Pharmaceutical University, GuangZhou
  • Yun Ma Experimental Animal Center of Sun Yan-sen University, GuangZhou
  • XinYan Deng Experimental Animal Center of Sun Yan-sen University, GuangZhou
  • GuangHui Cui Experimental Animal Center of Sun Yan-sen University, GuangZhou
  • Nan Gao Main Laboratory, ZhongShan Ophthalmic Center of Sun Yan-sen University, GuangZhou
  • Zhan Peng Main Laboratory, ZhongShan Ophthalmic Center of Sun Yan-sen University, GuangZhou

DOI:

https://doi.org/10.23675/sjlas.v33i3.107

Abstract

TheâIVS-2-654 C→T mutation accounts for approximately 20% of â thalassemia mutation in southern  China; it causes aberrant RNA splicing and leads to â thalassemia. To provide an animal model for testing  therapies for correcting splicing defects, we have produced two lines of transgenic mice with the human â  thalassemia mutant gene. The transgenic mice carrying this mutant gene show the same aberrant splicing  as their human counterparts and provide an animal model for testing therapies to correct splicing defects  at either the RNA or DNA level. 

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Published

01.12.2006

How to Cite

Lu, L., Huang, B., Chen, X., Wei, X., Yang, G., Ma, Y., Deng, X., Cui, G., Gao, N., & Peng, Z. (2006). A Transgenic Mouse Model for DNA/RNA Gene Therapy of Human β Thalassemia. Scandinavian Journal of Laboratory Animal Science, 33(3), 175–182. https://doi.org/10.23675/sjlas.v33i3.107

Issue

Vol. 33 No. 3 (2006)

Section

Articles