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A treatment for sickle cell disease in a mouse model

A treatment for sickle cell disease in a mouse model. Group A5 Laura Plank, Truc Nguyen, Joshua Trinidad, and Peyton Dent. Introduction . Sickle cell is an autosomal recessive genetic blood disorder with overdominance (Wikipedia).

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A treatment for sickle cell disease in a mouse model

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  1. A treatment for sickle cell disease in a mouse model Group A5 Laura Plank, Truc Nguyen, Joshua Trinidad, and Peyton Dent

  2. Introduction • Sickle cell is an autosomal recessive genetic blood disorder with overdominance (Wikipedia). • The disease is caused by an abnormal hemoglobin, hemoglobin S or HbS. • BCL11A is a gene that is found in humans and is also found in mice. • Gene silencing • Inactivating BCL11A http://www.childrenshospital.org/newsroom/Site1339/mainpageS1339P778.html

  3. Methods An experiment was setup to observe red blood cells of three different groups of mice. The first group was the control group The second group contained mice that had SCD The third group contained mice with SCD, but where BCL11A was inactivated.

  4. Data and Results (Xu, et.al.) http://www.sciencemag.org/content/334/6058/993/F1.large.jpg

  5. Data and Results (Xu, et.al.) http://www.sciencemag.org/content/334/6058/993/F1.large.jpg

  6. Data and Results (Xu, et.al.) http://www.sciencemag.org/content/334/6058/993/F3.expansion.html

  7. Discussion BCL11A is a transcription factor converting HbF to HbA Essential in erythroid maturation Silencing BCL11A prevents HbF conversion to HbA Absence of BCL11A does not affect erythroid maturity

  8. Conclusion Gene regulation is important in regards to reversing sickle cell disease through the silencing of BCL11A which reduces the phenotypic expression of SCD.

  9. Works Cited • Freeman, Scott. Biological Science. 4th ed. San Francisco, CA: Benjamin Cummings, 2011. Print • Graham, Rob. "Dialing up fetal hemoglobin dials down sickle cell disease." Boston Children's Hospital. N.p., 13 2011. Web. 19 Nov 2012.http://www.childrenshospital.org/newsroom/Site1339/mainpageS1339P778.html • “Sickle-cell disease.” Wikipedia. Web. 19 Nov. 2012. • Xu, Jian, et al. “Correction of Sickle Cell Disease in Adult Mice by Interference with Fetal Hemoglobin Silencing.” Science 334 (2011): 993-996. Web. 10 Nov. 2012.

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