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Characterization of In Vitro Models and Testing of RNAi Treatment for Achondroplasia

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Achondroplasia is a form of dwarfism caused by a conserved, Gly-Arg mutation in the transmembrane region of fibroblast growth factor receptor 3 (FGFR3). The goal was to infect different cell lines with lentivirus carrying shRNA to FGFR3, and quantify the knockdown of FGFR3 mRNA. To do this, establishment of a proper cell line was necessary. Mouse embryonic fibroblasts and C3H10T1/2 cells were differentiated for 12 days to determine maximal expression of FGFR3. The MEF cells were then infected with the lentiviral vector, and FGFR3 levels were again analyzed. Knockdown was observed, as expected, in the MEFs, with an overall decrease of 90% over uninfected. It thus appears that the shRNA, delivered by lentiviral vector, decreased the amount of FGFR3 being expressed.

  • This report represents the work of one or more WPI undergraduate students submitted to the faculty as evidence of completion of a degree requirement. WPI routinely publishes these reports on its website without editorial or peer review.
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  • E-project-043009-155943
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  • 2009
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  • 2009-04-30
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