Patisiran Pharmacokinetics, Pharmacodynamics, and Exposure-Response Analyses in the Phase 3 APOLLO Trial in Patients With Hereditary Transthyretin-Mediated (hATTR) Amyloidosis
Zhang X, Goel V, Attarwala H, Sweetser MT, Clausen VA, Robbie GJ. J Clin Pharmacol. 2019 Jul 19. PubMed
Multidomain Convergence of Argonaute during RISC Assembly Correlates with the Formation of Internal Water Clusters
Park MS, Araya-Secchi R, Brackbill JA, Phan HD, Kehling AC, Abd El-Wahab EW, Dayeh DM, Sotomayor M, Nakanishi K. Mol Cell. 2019 Jun 27. pii: S1097-2765(19)30444-7. PubMed
Strong and Sustained Antihypertensive Effect of Small Interfering RNA Targeting Liver Angiotensinogen
Uijl E, Mirabito Colafella KM, Sun Y, Ren L, van Veghel R, Garrelds IM, de Vries R, Poglitsch M, Zlatev I, Kim JB, Hoorn EJ, Foster D, Danser AHJ. Hypertension. 2019 Jun;73(6):1249-1257. PubMed
Investigational RNAi Therapeutic Targeting C5 Is Efficacious in Pre-clinical Models of Myasthenia Gravis
Kusner LL, Yucius K, Sengupta M, Sprague AG, Desai D, Nguyen T, Charisse K, Kuchimanchi S, Kallanthottathil R, Fitzgerald K, Kaminski HJ, Borodovsky A. Mol Ther Methods Clin Dev. 2019 May 10;13:484-492. PubMed
Delivery of GalNAc-Conjugated Splice-Switching ASOs to Non-hepatic Cells through Ectopic Expression of Asialoglycoprotein Receptor
Scharner J, Qi S, Rigo F, Bennett CF, Krainer AR. Mol Ther Nucleic Acids. 2019 Jun 7;16:313-325. PubMed
Delivery of Cell-Specific Aptamers to the Arterial Wall with an Occlusion Perfusion Catheter
Udofot O, Lin LH, Thiel WH, Erwin M, Turner E, Miller FJ Jr, Giangrande PH, Yazdani SK. Mol Ther Nucleic Acids. 2019 Jun 7;16:360-366. PubMed
Use of “C9/11 Mismatch” Control Small Interfering RNA Reveals Sequence-Related Off-Target Effect on Coagulation of an Small Interfering RNA Targeting Mouse Coagulation Factor XII
Heestermans M, de Jong A, van Tilburg S, Reitsma PH, Versteeg HH, Spronk HM, van Vlijmen BJM. Nucleic Acid Ther. 2019 May 7. PubMed
LncRNA SNHG10 facilitates hepatocarcinogenesis and metastasis by modulating its homolog SCARNA13 via a positive feedback loop
Lan T, Yuan K, Yan X, Xu L, Liao H, Hao X, Wang J, Liu H, Xie K, Li J, Liao M, Huang J, Zeng Y, Wu H. Cancer Res. 2019 May 17. PubMed
Effect of Sugar 2′,4′-Modifications on Gene Silencing Activity of Small Interfering RNA Duplexes
Malek-Adamian E, Fakhoury J, Arnold AE, MartĂnez-Montero S, Shoichet MS, Damha MJ. Nucleic Acid Ther. 2019 May 14. PubMed
5′-Morpholino modification of the sense strand of an siRNA makes it a more effective passenger
Kumar P, Parmar RG, Brown CR, Willoughby JLS, Foster DJ, Babu IR, Schofield S, Jadhav V, Charisse K, Nair JK, Rajeev KG, Maier MA, Egli M, Manoharan M. Chem Commun (Camb). 2019 Apr 12. PubMed