A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system

Paper of the MonthBy OTSSeptember 29, 2019Leave a comment

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, Sena-Esteves M, Pai AA, DiFiglia M, Aronin N, Khvorova A. Nat…

Allele-selective transcriptional repression of mutant HTT for the treatment of Huntington’s disease

Paper of the MonthBy OTSAugust 21, 2019Leave a comment

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, Laganiere J, Vu BJ, Narayanan A, Sedaghat Y, Tillack K, Thiede…

Traceless aptamer-mediated isolation of CD8+ T cells for chimeric antigen receptor T-cell therapy

Paper of the MonthBy OTSJuly 24, 2019Leave a comment

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 have shown excellent results in clinical trials and two were approved by the FDA for clinical use in 2017 (Kymriah and Yescarta).…

Targeting Huntingtin Expression in Patients with Huntington’s Disease

Paper of the MonthBy OTSJune 26, 2019Leave a comment

Tabrizi SJ, Leavitt BR, Landwehrmeyer GB, Wild EJ, Saft C, Barker RA, Blair NF, Craufurd D, Priller J, Rickards H, Rosser A, Kordasiewicz HB, Czech C, Swayze EE, Norris DA, Baumann T, Gerlach I, Schobel SA, Paz E, Smith AV, Bennett CF, Lane RM. N Engl J Med. 2019 May 6 The success of nusinersen…