January 2021 Paper of the Month
Koblan LW, Erdos MR, Wilson C, Cabral WA, Levy JM, Xiong ZM, Tavarez UL, Davison LM, Gete YG, Mao X, Newby GA, Doherty SP, Narisu N, Sheng Q, Krilow C, Lin CY, Gordon LB, Cao K, Collins FS, Brown JD, Liu DR. Nature. 2021 Jan;589(7843):608-614.
Joint December 2020 Papers of the Month
Polack FP, Thomas SJ, Kitchin N, Absalon J, Gurtman A, Lockhart S, Perez JL, Pérez Marc G, Moreira ED, Zerbini C, Bailey R, Swanson KA, Roychoudhury S, Koury K, Li P, Kalina WV, Cooper D, Frenck RW Jr, Hammitt LL, Türeci Ö, Nell H, Schaefer A, Ünal S, Tresnan DB, Mather S, Dormitzer PR, Şahin U, Jansen KU, Gruber WC; C4591001 Clinical Trial Group.
November 2020 Paper of the Month
Building on previous work, here Asami, Nagata, Yoshioka and colleagues from the Tokyo Medical and Dental University and Ionis Pharmaceuticals investigate new heteroduplex oligonucleotide (HDO) designs for antisense oligonucleotides.
October 2020 Paper of the Month
The chromosomal 2q21.3 locus has undergone positive selection in the last 5000 years in Europeans and this has long been attributed to the presence of the lactase gene.
September 2020 Paper of the Month
Non-alcoholic steatohepatitis (NASH) is caused by accumulation of fat in the liver which can result in liver inflammation, cirrhosis and subsequent liver failure.
August 2020 Joint Papers of the Month
Antisense oligonucleotide modulation of non-productive alternative splicing upregulates gene ...
July 2020 Joint Papers of the Month
Phase 1/2 study of COVID-19 RNA vaccine BNT162b1 in ...
Reversing a Model of Parkinson’s Disease With in Situ Converted Nigral Neurons
Qian H, Kang X, Hu J, Zhang D, Liang ...
Base-pair Conformational Switch Modulates miR-34a Targeting of Sirt1 mRNA
Baronti L, Guzzetti I, Ebrahimi P, Friebe Sandoz S, Steiner ...
Imaging small molecule-induced endosomal escape of siRNA
Du Rietz H, Hedlund H, Wilhelmson S, Nordenfelt P, ...
March 2020 Joint Papers of the Month
Inclisiran for the Treatment of Heterozygous Familial Hypercholesterolemia. Raal ...
A native function for RAN translation and CGG repeats in regulating fragile X protein synthesis
Rodriguez CM, Wright SE, Kearse MG, Haenfler JM, Flores BN, ...
January 2020 Joint Papers of the Month
Directed RNase H Cleavage of Nascent Transcripts Causes Transcription Termination ...
mRNA Structure Regulates Protein Expression Through Changes in Functional Half-Life
Mauger DM, Cabral BJ, Presnyak V, Su SV, Reid ...
Nusinersen Initiated in Infants During the Presymptomatic Stage of Spinal Muscular Atrophy
De Vivo DC, Bertini E, Swoboda KJ, Hwu WL, ...
Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease
Kim J, Hu C, Moufawad El Achkar C, Black ...
Peptide-conjugated oligonucleotides evoke long-lasting myotonic dystrophy correction in patient-derived cells and mice
Klein AF, Varela MA, Arandel L, Holland A, Naouar ...
A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system
Alterman JF, Godinho BMDC, Hassler MR, Ferguson CM, Echeverria ...
Allele-selective transcriptional repression of mutant HTT for the treatment of Huntington’s disease
Zeitler B, Froelich S, Marlen K, Shivak DA, Yu ...
Traceless aptamer-mediated isolation of CD8+ T cells for chimeric antigen receptor T-cell therapy
Nataly Kacherovsky and Ian Cardle Kacherovsky N, ...
Targeting Huntingtin Expression in Patients with Huntington’s Disease
Tabrizi SJ, Leavitt BR, Landwehrmeyer GB, Wild EJ, Saft ...
Guidelines for Experiments Using Antisense Oligonucleotides and Double-Stranded RNAs
Gagnon KT, Corey DR. Nucleic Acid Ther. 2019 Mar ...
Safety evaluation of 2′-deoxy-2′-fluoro nucleotides in GalNAc-siRNA conjugates
Janas MM, Zlatev I, Liu J, Jiang Y, Barros SA, ...
Phase 1 Trial of an RNA Interference Therapy for Acute Intermittent Porphyria
Sardh E, Harper P, Balwani M, Stein P, Rees D, ...
RNA inhibitors of nuclear proteins responsible for multiple organ dysfunction syndrome
Urak KT, Blanco GN, Shubham S, Lin LH, Dassie JP, ...
Combination of aptamer and drug for reversible anticoagulation in cardiopulmonary bypass
Gunaratne R, Kumar S, Frederiksen JW, Stayrook S, Lohrmann JL, ...
Reversal of siRNA-mediated gene silencing in vivo
Zlatev I, Castoreno A, Brown CR, Qin J, Waldron S, ...
Antisense oligonucleotides correct the familial dysautonomia splicing defect in IKBKAP transgenic mice
Sinha R, Kim YJ, Nomakuchi T, Sahashi K, Hua Y, ...
Gene activation of CEBPA using saRNA: preclinical studies of the first in human saRNA drug candidate for liver cancer
Reebye V, Huang KW, Lin V, Jarvis S, Cutilas P, ...
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, ...
Eradication of spontaneous malignancy by local immunotherapy
Sagiv-Barfi I, Czerwinski DK, Levy S, Alam IS, Mayer AT, ...
Systemic Messenger RNA Therapy as a Treatment for Methylmalonic Acidemia
An D, Schneller JL, Frassetto A, Liang S, Zhu X, ...
Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy
Finkel RS, Mercuri E, Darras BT, Connolly AM, Kuntz NL, ...
Nucleic acid binding proteins affect the subcellular distribution of phosphorothioate antisense oligonucleotides
Bailey JK, Shen W, Liang XH, Crooke ST. Nucleic Acids ...
