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Song D, Dunaief JL

This modification specifically binds to the ARG1 gene promoter to drive its expression, a process significantly inhibited by GNE-140 (LDHA inhibitor) or isosafrole (123)

Neurobiol Dis 77:4961

This approach ensures the cleavage at the specific sites of mutant but not of normal CFTR alleles by taking advantage of different techniques as the CRISP/Cas9 and specific RNA guides

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