Leading RNA Interference Therapeutics Part 1: Silencing Hereditary Transthyretin Amyloidosis, with a Focus on Patisiran

Titze-de-Almeida, SS; Brandao, PRD; Faber, I; Titze-de-Almeida, R

Titze-de-Almeida, R (reprint author), Univ Brasilia, Technol Gene Therapy Lab, Cent Inst Sci, FAV, Brasilia, DF, Brazil.

MOLECULAR DIAGNOSIS & THERAPY, 0; ():

Abstract

In 2018, patisiran was the first-ever RNA interference (RNAi)-based drug approved by the US Food and Drug Administration. Now pharmacology textbooks m......

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