A native function for RAN translation and CGG repeats in regulating fragile X protein synthesis

March 26th, 2020|Categories: Paper of the Month|

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. ...

Nusinersen Initiated in Infants During the Presymptomatic Stage of Spinal Muscular Atrophy

December 19th, 2019|Categories: Paper of the Month|

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 ...

Peptide-conjugated oligonucleotides evoke long-lasting myotonic dystrophy correction in patient-derived cells and mice

November 2nd, 2019|Categories: Paper of the Month|

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

September 29th, 2019|Categories: Paper of the Month|

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

August 21st, 2019|Categories: Paper of the Month|

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

July 24th, 2019|Categories: Paper of the Month|

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 ...

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