Team:NJU-China

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New Strategy for Treating

Fragile X Syndrome

by Targeting Expression of

FMRP Using TBEV5'-UTR

———— NJU-CHINA

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We (NJU-China 2018 team) aim to find the possible therapy of the Fragile X Syndrome, a genetical disease causing a great number of ill of brain. As is previously analyzed, the syndrome is due to the reduction of a specific protein named FMR-1 in the dendrites of our neural cells. In order to innovate the treatment, we developed a strategy by using a designed sequence of mRNA to transfer the target gene specially into the dendrites for higher efficiency and accuracy. With the production of the certain sequence, we are able to maintain the production of certain protein in the neuron to the required level.