The chemical structure and phosphorothioate content of hydrophobically modified siRNAs impact extrahepatic distribution and efficacy.
Biscans A, Caiazzi J, Davis S, McHugh N, Sousa J, Khvorova A. Nucleic Acids Res. 2020 Jul 16 Pubmed
G4C2 Repeat RNA Initiates a POM121-Mediated Reduction in Specific Nucleoporins in C9orf72 ALS/FTD
Coyne AN, Zaepfel BL, Hayes L, Fitchman B, Salzberg Y, Luo EC, Bowen K, Trost H, Aigner S, Rigo F, Yeo GW, Harel A, Svendsen CN, Sareen D, Rothstein JD. Neuron. 2020 Jul 8:S0896-6273(20)30481-5. Pubmed
Suppression of proteolipid protein rescues Pelizaeus-Merzbacher disease
Elitt MS, Barbar L, Shick HE, Powers BE, Maeno-Hikichi Y, Madhavan M, Allan KC, Nawash BS, Gevorgyan AS, Hung S, Nevin ZS, Olsen HE, Hitomi M, Schlatzer DM, Zhao HT, Swayze A, LePage DF, Jiang W, Conlon RA, Rigo F, Tesar PJ. Nature. 2020 Jul ...
Interview with Christian Berk, PhD
Christian Berk, Ph.D., ETH Zurich How did you become interested in the field of oligonucleotides? Frankly, I did not know much about RNA therapeutics when I joined Jonathan Hall’s group at ETH Zurich. After finishing my studies, I just knew that I wanted to ...
Reversing a Model of Parkinson’s Disease With in Situ Converted Nigral Neurons
Qian H, Kang X, Hu J, Zhang D, Liang Z, Meng F, Zhang X, Xue Y, Maimon R, Dowdy SF, Devaraj NK, Zhou Z, Mobley WC, Cleveland DW, Fu XD. Nature. 2020 Jun;582(7813):550-556. Joint First Authors Dr. Hao Qian Parkinson’s ...
Therapeutic siRNA: state of the art
Hu B, Zhong L, Weng Y, Peng L, Huang Y, Zhao Y, Liang XJ. Signal Transduct Target Ther. 2020 Jun 19;5(1):101. Pubmed
Delivery of Oligonucleotides to the Liver with GalNAc: From Research to Registered Therapeutic Drug
Debacker AJ, Voutila J, Catley M, Blakey D, Habib N. Mol Ther. 2020 Jun 17:S1525-0016(20)30305-1. Pubmed
Phosphorothioate modified oligonucleotide-protein interactions
Crooke ST, Vickers TA, Liang XH. Nucleic Acids Res. 2020 Jun 4;48(10):5235-5253. Pubmed
Rational design of aptamer switches with programmable pH response
Thompson IAP, Zheng L, Eisenstein M, Soh HT. Nat Commun. 2020 Jun 10;11(1):2946. Pubmed
Determination of RNA structural diversity and its role in HIV-1 RNA splicing
Tomezsko PJ, Corbin VDA, Gupta P, Swaminathan H, Glasgow M, Persad S, Edwards MD, Mcintosh L, Papenfuss AT, Emery A, Swanstrom R, Zang T, Lan TCT, Bieniasz P, Kuritzkes DR, Tsibris A, Rouskin S. Nature. 2020 Jun;582(7812):438-442. Pubmed