MTL-CEBPA, a small activating RNA therapeutic up-regulating C/EBP-α, in patients with advanced liver cancer: a first-in-human, multi-centre, open-label, phase I trial
Sarker D, Plummer R, Meyer T, Sodergren M, Basu B, Chee CE, Huang KW, Palmer DH, Ma YT, Evans TRJ, Spalding DR, Pai M, Sharma R, Pinato DJ, Spicer J, Hunter S, Kwatra V, Nicholls JP, Collin D, Nutbrown R, Glenny H, Fairbairn S, Reebye ...
Treatment of Hemophilia A Using Factor VIII Messenger RNA Lipid Nanoparticles
Chen CY, Tran DM, Cavedon A, Cai X, Rajendran R, Lyle MJ, Martini PGV, Miao CH. Mol Ther Nucleic Acids. 2020 Apr 7;20:534-544 PubMed
Engineered Circular RNA Sponges Act as miRNA Inhibitors to Attenuate Pressure Overload-Induced Cardiac Hypertrophy
Lavenniah A, Luu TDA, Li YP, Lim TB, Jiang J, Ackers-Johnson M, Foo RS. Mol Ther. 2020 Apr 14. pii: S1525-0016(20)30187-8. PubMed
Genomic Clustering Facilitates Nuclear Processing of Suboptimal Pri-miRNA Loci
Shang R, Baek SC, Kim K, Kim B, Kim VN, Lai EC. Mol Cell. 2020 Apr 16;78(2):303-316.e4. PubMed
MicroRNA Clustering Assists Processing of Suboptimal MicroRNA Hairpins through the Action of the ERH Protein
Fang W, Bartel DP. Mol Cell. 2020 Apr 16;78(2):289-302.e6. PubMed
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: Patrik Andersson (AstraZeneca) Date: May 13, 2020 Description: 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
