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. ...
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, Liu Y, Ifrim MF, Glineburg MR, Krans A, Jafar-Nejad P, Sutton MA, Bassell GJ, Parent JM, Rigo F, Barmada SJ, Todd PK. Nat Neurosci. 2020 Mar;23(3):386-397. Joint First Authors Prof. Peter K. ...
January 2020 Joint Papers of the Month
Directed RNase H Cleavage of Nascent Transcripts Causes Transcription Termination Lai F, Damle SS, Ling KK, Rigo F. Mol Cell. 2020 Jan 3. pii: S1097-2765(19)30959-1. Joint First Authors Antisense-Mediated Transcript Knockdown Triggers Premature Transcription Termination. Lee JS, Mendell JT. Mol ...
mRNA Structure Regulates Protein Expression Through Changes in Functional Half-Life
Mauger DM, Cabral BJ, Presnyak V, Su SV, Reid DW, Goodman B, Link K, Khatwani N, Reynders J, Moore MJ, McFadyen IJ. Proc Natl Acad Sci U S A. 2019 Nov 26;116(48):24075-24083. Melissa J. Moore Iain J. McFadyen ...
Nusinersen Initiated in Infants During the Presymptomatic Stage of Spinal Muscular Atrophy
De Vivo DC, Bertini E, Swoboda KJ, Hwu WL, Crawford TO, Finkel RS, Kirschner J, Kuntz NL, Parsons JA, Ryan MM, Butterfield RJ, Topaloglu H, Ben-Omran T, Sansone VA, Jong YJ, Shu F, Staropoli JF, Kerr D, Sandrock AW, Stebbins C, Petrillo M, Braley ...
Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease
Kim J, Hu C, Moufawad El Achkar C, Black LE, Douville J, Larson A, Pendergast MK, Goldkind SF, Lee EA, Kuniholm A, Soucy A, Vaze J, Belur NR, Fredriksen K, Stojkovska I, Tsytsykova A, Armant M, DiDonato RL, Choi J, Cornelissen L, Pereira LM, ...
Peptide-conjugated oligonucleotides evoke long-lasting myotonic dystrophy correction in patient-derived cells and mice
Klein AF, Varela MA, Arandel L, Holland A, Naouar N, Arzumanov A, Seoane D, Revillod L, Bassez G, Ferry A, Jauvin D, Gourdon G, Puymirat J, Gait MJ, Furling D, Wood MJ. J Clin Invest. 2019 Sep 30. Joint First Authors Klein ...
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 D, Sapp E, Haraszti RA, Coles AH, Conroy F, Miller R, Roux L, Yan P, Knox EG, Turanov AA, King RM, Gernoux G, Mueller C, Gray-Edwards HL, Moser RP, Bishop NC, Jaber SM, Gounis MJ, ...
Allele-selective transcriptional repression of mutant HTT for the treatment of Huntington’s disease
Zeitler B, Froelich S, Marlen K, Shivak DA, Yu Q, Li D, Pearl JR, Miller JC, Zhang L, Paschon DE, Hinkley SJ, Ankoudinova I, Lam S, Guschin D, Kopan L, Cherone JM, Nguyen HB, Qiao G, Ataei Y, Mendel MC, Amora R, Surosky R, ...
Traceless aptamer-mediated isolation of CD8+ T cells for chimeric antigen receptor T-cell therapy
Nataly Kacherovsky and Ian Cardle Kacherovsky N, Cardle II, Cheng EL, Yu JL, Baldwin ML, Salipante SJ, Jensen MC, Pun SH. Nat Biomed Eng. 2019 Jun 17. Genetically engineered chimeric antigen receptor (CAR) T-cell therapies for liquid cancers ...
