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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Laura Plank, Truc Nguyen, Joshua Trinidad, and Peyton Dent
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.
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
Gene regulation is important in regards to reversing sickle cell disease through the silencing of BCL11A which reduces the phenotypic expression of SCD.