Leading RNA Interference Therapeutics Part 1: Silencing Hereditary Transthyretin Amyloidosis, with a Focus on Patisiran.
作者： Simoneide S. Titze-de-Almeida , Pedro Renato de Paula Brandão , Ingrid Faber , Ricardo Titze-de-Almeida
DOI: 10.1007/S40291-019-00434-W
关键词: Gene knockdown 、 Cmax 、 MRNA cleavage 、 Amyloidosis 、 Medicine 、 RNA interference 、 Gene silencing 、 Pharmacology 、 Transthyretin 、 Small interfering RNA
摘要: … The use of cationic lipids to transfect IV injected siRNAs into hepatocytes were first described in non-human primates [38]. In the formulation of patisiran, the lipid particles are first …
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