GalNAc-siRNA Conjugates: Leading the Way for Delivery of RNAi Therapeutics
Springer AD, Dowdy SF. Nucleic Acid Ther. 2018 Jun;28(3):109-118. PubMed
Lipid Nanoparticles Enabling Gene Therapies: From Concepts to Clinical Utility
Kulkarni JA, Cullis PR, van der Meel R. Nucleic Acid Ther. 2018 Jun;28(3):146-157. PubMed
Current Status of Messenger RNA Delivery Systems
Stanton MG. Nucleic Acid Ther. 2018 Jun;28(3):158-165. PubMed
Intracellular Trafficking and Endosomal Release of Oligonucleotides: What We Know and What We Don’t
Juliano RL. Nucleic Acid Ther. 2018 Jun;28(3):166-177. PubMed
Aptamer-Mediated Delivery and Cell-Targeting Aptamers: Room for Improvement
Yan AC, Levy M. Nucleic Acid Ther. 2018 Jun;28(3):194-199. PubMed
Circular RNAs in Cancer – Lessons Learned From microRNAs
Dragomir M, Calin GA. Front Oncol. 2018 May 28;8:179. PubMed
A Single Administration of CRISPR/Cas9 Lipid Nanoparticles Achieves Robust and Persistent In Vivo Genome Editing
Finn JD, Smith AR, Patel MC, Shaw L, Youniss MR, van Heteren J, Dirstine T, Ciullo C, Lescarbeau R, Seitzer J, Shah RR, Shah A, Ling D, Growe J, Pink M, Rohde E, Wood KM, Salomon WE, Harrington WF, Dombrowski C, Strapps WR, Chang Y, ...
Oligonucleotide Therapeutics: Technology Evolution for Clinically Relevant Tissue Delivery
Presenter: Anastasia Khvorova, PhD, RNA Therapeutics Institute, University of Massachusetts Medical School Date: 10 September 2018 Description: A decade of progress in oligonucleotide chemistry and formulation development has resulted in several compounds, both siRNAs and antisense, demonstrating robust clinical activity. Based on sequence, these ...
Identifying and avoiding off-target effects of RNase H-dependent antisense oligonucleotides in mice
Hagedorn PH, Pontoppidan M, Bisgaard TS, Berrera M, Dieckmann A, Ebeling M, Møller MR, Hudlebusch H, Jensen ML, Hansen HF, Koch T, Lindow M. Nucleic Acids Res. 2018 May 22. PubMed
Therapeutic silencing of FSP27 reduces the progression of atherosclerosis in Ldlr-/- mice
Rajamoorthi A, Lee RG, Baldán Á. Atherosclerosis. 2018 May 24;275:43-49. PubMed
