CRISPR-Based Therapeutic Genome Editing: Strategies and In Vivo Delivery by AAV Vectors
Wang D, Zhang F, Gao G. Cell. 2020 Apr 2;181(1):136-150. PubMed
March 2020 Joint Papers of the Month
Inclisiran for the Treatment of Heterozygous Familial Hypercholesterolemia. Raal FJ, Kallend D, Ray KK, Turner T, Koenig W, Wright RS, Wijngaard PLJ, Curcio D, Jaros MJ, Leiter LA, Kastelein JJP; ORION-9 Investigators. N Engl J Med. 2020 Mar 18. Prof. Frederick J. ...
Safety of Therapeutic Oligonucleotides
Presenter:Â Patrik Andersson (AstraZeneca) Date: May 13, 2020 Description:Â Successful therapeutics need to be efficacious and safe. Although oligonucleotide therapeutics act via unique mechanisms, they follow the same principles for safety assessment as other modalities. This webinar will give an overview of these principles and what ...
An Antisense Oligonucleotide Leads to Suppressed Transcription of Hdac2 and Long-Term Memory Enhancement
Poplawski SG, Garbett KA, McMahan RL, Kordasiewicz HB, Zhao H, Kennedy AJ, Goleva SB, Sanders TH, Motley ST, Swayze EE, Ecker DJ, Sweatt JD, Michael TP, Greer CB. Mol Ther Nucleic Acids. 2020 Mar 6;19:1399-1412. PubMed
Chemical Diversity of Locked Nucleic Acid-Modified Antisense Oligonucleotides Allows Optimization of Pharmaceutical Properties
Papargyri N, Pontoppidan M, Andersen MR, Koch T, Hagedorn PH. Mol Ther Nucleic Acids. 2020 Mar 6;19:706-717. PubMed
Likelihood of Nonspecific Activity of Gapmer Antisense Oligonucleotides Is Associated with Relative Hybridization Free Energy
Watt AT, Swayze G, Swayze EE, Freier SM. Nucleic Acid Ther. 2020 Mar 3. PubMed
Chimeric siRNAs with chemically modified pentofuranose and hexopyranose nucleotides: altritol-nucleotide (ANA) containing GalNAc-siRNA conjugates: in vitro and in vivo RNAi activity and resistance to 5′-exonuclease
Kumar P, Degaonkar R, Guenther DC, Abramov M, Schepers G, Capobianco M, Jiang Y, Harp J, Kaittanis C, Janas MM, Castoreno A, Zlatev I, Schlegel MK, Herdewijn P, Egli M, Manoharan M. Nucleic Acids Res. 2020 Mar 14. PubMed
U1 snRNP regulates chromatin retention of noncoding RNAs
Yin Y, Lu JY, Zhang X, Shao W, Xu Y, Li P, Hong Y, Cui L, Shan G, Tian B, Zhang QC, Shen X. Nature. 2020 Apr;580(7801):147-150. PubMed
Endogenous MicroRNA Competition as a Mechanism of shRNA-Induced Cardiotoxicity
Course MM, Gudsnuk K, Desai N, Chamberlain JR, Valdmanis PN. Mol Ther Nucleic Acids. 2020 Mar 6;19:572-580. PubMed
Extracellular nanovesicles for packaging of CRISPR-Cas9 protein and sgRNA to induce therapeutic exon skipping
Gee P, Lung MSY, Okuzaki Y, Sasakawa N, Iguchi T, Makita Y, Hozumi H, Miura Y, Yang LF, Iwasaki M, Wang XH, Waller MA, Shirai N, Abe YO, Fujita Y, Watanabe K, Kagita A, Iwabuchi KA, Yasuda M, Xu H, Noda T, Komano J, Sakurai ...
