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it is a powerful gene-modulating agent with profound systemic and topical applications

In another embodiment the ligand is a ligand that is endocytosed and/or transcytosed into and/or through the target cell as may be assayed by a cell culture model of the BBB (using primary isolated bovine brain capillary endothelial cells (BCEC)) as described by Gaillard et al

Foot NJ, Leong YA, Dorstyn LE, Dalton HE, Ho K, Zhao L, et al

What human clinical trial data exists to inform dosing and safety expectations

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Knocking down the two genes by siRNA also confirmed the sensitivity of G12D and G12V mutant cells to the inhibition of GLS and GS together (Appendix Fig