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In the context of FXS, instead of targeting NATs of the FMR1 gene, there has been success in restoring FMR1 expression in induced pluripotent stem cells (iPSCs) through direct CRISPR-Cas9-mediated deletion of pathological repeat sequences of the sense gene [61]
TB-500 Dosing Math by Vial Size 5 mg vial + 1 mL bacteriostatic water = 5 mg/mL (5,000 mcg/mL) 5 mg vial + 2 mL bacteriostatic water = 2.5 mg/mL (2,500 mcg/mL) 10 mg vial + 2 mL bacteriostatic water = 5 mg/mL (5,000 mcg/mL) The 5 mg/mL ratio (1 mL of water per 5 mg vial) is the most popular setup because the doses land on clean, easy numbers
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