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Randomized Phase 2 Study of GMI-1070 in SCD: Reduction in Time to Resolution of Vaso-Occlusive Events and Decreased Opioid Use

doi: 10.1038/cgt.2013.56 161 Pacheco-OrozcoRAMontealegre-PezLCayolFMartnez-GregorioHOliverJFrechaCet al

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To overcome these barriers, future research must prioritize the advancement of delivery technologies and the enhancement of mRNA construct stability at the molecular level