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Nusinersen: first global approval

PMID: 164 ZhuXBiCCaoWLiSYuanCXuPet al

P.RennerK.BishopD

doi: 10.3390/pharmaceutics15122766 25 El MehdiIFalcoSIBoujrafSMustaphaHCamposMGVilas-BoasM

10.1016/j.eplepsyres.2020.106499 21

The removal of the intestinal GK gene, and the LSL cassette ahead of the introduced GK and its mutants (to trigger the expression of introduced GK), was then achieved by intraperitoneal injection of tamoxifen