Synthesis, chirality-dependent conformational and biological properties of siRNAs containing 5′-(R)- and 5′-(S)-C-methyl-guanosine
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Antisense Drug Discovery and Development Technology Considered in a Pharmacological Context
Crooke ST, Liang XH, Crooke RM, Baker BF, Geary RS. Biochem Pharmacol. 2020 Aug 12:114196. Pubmed
Single Dose Pharmacokinetics and Pharmacodynamics of Transthyretin Targeting GalNAc-siRNA Conjugate, Vutrisiran, in Healthy Subjects.
Habtemariam BA, Karsten V, Attarwala H, Goel V, Melch M, Clausen VA, Garg P, Vaishnaw AK, Sweetser MT, Robbie GJ, Vest J. Clin Pharmacol Ther. 2020 Jun 29. Pubmed
Therapeutic siRNA: state of the art
Hu B, Zhong L, Weng Y, Peng L, Huang Y, Zhao Y, Liang XJ. Signal Transduct Target Ther. 2020 Jun 19;5(1):101. Pubmed
Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria
Balwani M, Sardh E, Ventura P, Peiró PA, Rees DC, Stölzel U, Bissell DM, Bonkovsky HL, Windyga J, Anderson KE, Parker C, Silver SM, Keel SB, Wang JD, Stein PE, Harper P, Vassiliou D, Wang B, Phillips J, Ivanova A, Langendonk JG, Kauppinen R, Minder ...
Emerging siRNA Design Principles and Consequences for Biotransformation and Disposition in Drug Development
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