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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...

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Detalles Bibliográficos
Autores principales: Majeed, Chaudry Nasir, Ma, Christopher D, Xiao, Ted, Rudnick, Sean, Bonkovsky, Herbert L
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Dove 2022
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