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Spotlight on Givosiran as a Treatment Option for Adults with Acute Hepatic Porphyria: Design, Development, and Place in Therapy
Small interfering ribonucleic acids [siRNAs] are short ribonucleic acid (RNA) fragments cleaved from double-stranded RNA molecules that target and bind to specific sequences on messenger RNA (mRNA), leading to their destruction. Therefore, the siRNA down-regulates the formation of selected mRNAs and...
Autores principales: | Majeed, Chaudry Nasir, Ma, Christopher D, Xiao, Ted, Rudnick, Sean, Bonkovsky, Herbert L |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Dove
2022
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9208469/ https://www.ncbi.nlm.nih.gov/pubmed/35734365 http://dx.doi.org/10.2147/DDDT.S281631 |
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