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doi: 10.1016/j.bbalip.2004.03.006
Transposons can carry foreign genes to leapfrog integrate in the genome
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According to the phase 1 clinical data disclosed by Alnylam, 9 givosiran dosed at 2.5 mg/kg once-monthly significantly reduced the levels of ALAS1 mRNA, delta aminolevulinic acid (ALA) and porphobilinogen (PBG), with higher than 80% inhibition efficiency, which was accomplished with a 79% decrease in the mean annualized attack rate compared to that of the placebo
