Targeting Huntingtin Expression in Patients with Huntington’s Disease
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. ...
Guidelines for Experiments Using Antisense Oligonucleotides and Double-Stranded RNAs
Gagnon KT, Corey DR. Nucleic Acid Ther. 2019 Mar 23. Keith T. Gagnon David Corey In addition to the widespread use of synthetic oligonucleotides to manipulate RNA levels in basic research, interest in oligonucleotide ...
Safety evaluation of 2′-deoxy-2′-fluoro nucleotides in GalNAc-siRNA conjugates
Janas MM, Zlatev I, Liu J, Jiang Y, Barros SA, Sutherland JE, Davis WP, Liu J, Brown CR, Liu X, Schlegel MK, Blair L, Zhang X, Das B, Tran C, Aluri K, Li J, Agarwal S, Indrakanti R, Charisse K, Nair J, Matsuda S, Rajeev ...
Phase 1 Trial of an RNA Interference Therapy for Acute Intermittent Porphyria
Sardh E, Harper P, Balwani M, Stein P, Rees D, Bissell DM, Desnick R, Parker C, Phillips J, Bonkovsky HL, Vassiliou D, Penz C, Chan-Daniels A, He Q, Querbes W, Fitzgerald K, Kim JB, Garg P, Vaishnaw A, Simon AR, Anderson KE. N Engl J ...
RNA inhibitors of nuclear proteins responsible for multiple organ dysfunction syndrome
Urak KT, Blanco GN, Shubham S, Lin LH, Dassie JP, Thiel WH, Chen Y, Sonkar VK, Lei B, Murthy S, Gutierrez WR, Wilson ME, Stiber JA, Klesney-Tait J, Dayal S, Miller FJ Jr, Giangrande PH. Nat Commun. 2019 Jan 10;10(1):116. The three joint first authors: ...
Combination of aptamer and drug for reversible anticoagulation in cardiopulmonary bypass
Gunaratne R, Kumar S, Frederiksen JW, Stayrook S, Lohrmann JL, Perry K, Bompiani KM, Chabata CV, Thalji NK, Ho MD, Arepally G, Camire RM, Krishnaswamy S, Sullenger BA. Nat Biotechnol. 2018 Aug;36(7):606-613. The two joint first authors: Ruwan Gunaratne Kumar Shekhar ...
Reversal of siRNA-mediated gene silencing in vivo
Zlatev I, Castoreno A, Brown CR, Qin J, Waldron S, Schlegel MK, Degaonkar R, Shulga-Morskaya S, Xu H, Gupta S, Matsuda S, Akinc A, Rajeev KG, Manoharan M, Maier MA, Jadhav V. Nat Biotechnol. 2018 Jul;36(6):509-511. Clinical data from GalNAc-conjugated siRNAs demonstrate long-term persistence of ...
Antisense oligonucleotides correct the familial dysautonomia splicing defect in IKBKAP transgenic mice
Sinha R, Kim YJ, Nomakuchi T, Sahashi K, Hua Y, Rigo F, Bennett CF, Krainer AR. Nucleic Acids Res. 2018 Jun 1;46(10):4833-4844. The three joint first authors: Rahul Sinha Young Jin Kim Tomoki Nomakuchi Currently, the most successful oligonucleotide ...
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, Dorman S, Ciriello S, Andrikakou P, Voutila J, Saetrom P, Mintz PJ, Reccia I, Rossi JJ, Huber H, Habib R, Kostomitsopoulos N, Blakey DC, Habib NA. Oncogene. 2018 Jun;37(24):3216-3228 Small activating RNAs (saRNA) are the ...
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, van Heteren J, Dirstine T, Ciullo C, Lescarbeau R, Seitzer J, Shah RR, Shah A, Ling D, Growe J, Pink M, Rohde E, Wood KM, Salomon WE, Harrington WF, Dombrowski C, Strapps WR, Chang Y, ...
