Tilsotolimod with ipilimumab drives tumor responses in anti-PD-1 refractory melanoma
Papers of interest to society members in the field of oligonucleotide therapeutics. Antisense, aptamers, lncRNA, siRNA, splice modulation and ribozymes.
Super-resolution RNA imaging using a rhodamine-binding aptamer with fast exchange kinetics
Sunbul M, Lackner J, Martin A, Englert D, Hacene B, Grün F, Nienhaus K, Nienhaus GU, Jäschke A. Nat Biotechnol. 2021 Feb 11. Pubmed
Intrastriatal Administration of AAV5-miHTT in Non-Human Primates and Rats Is Well Tolerated and Results in miHTT Transgene Expression in Key Areas of Huntington Disease Pathology
Spronck EA, Vallès A, Lampen MH, Montenegro-Miranda PS, Keskin S, Heijink L, Evers MM, Petry H, Deventer SJV, Konstantinova P, Haan M. Brain Sci. 2021 Jan 20;11(2):E129. Pubmed .
Ligand conjugated antisense oligonucleotide for the treatment of transthyretin amyloidosis: preclinical and phase 1 data
Viney NJ, Guo S, Tai LJ, Baker BF, Aghajan M, Jung SW, Yu RZ, Booten S, Murray H, Machemer T, Burel S, Murray S, Buchele G, Tsimikas S, Schneider E, Geary RS, Benson MD, Monia BP. ESC Heart Fail. 2020 Dec 7. Pubmed ...
Post-Transcriptional Genetic Silencing of BCL11A to Treat Sickle Cell Disease
Esrick EB, Lehmann LE, Biffi A, Achebe M, Brendel C, Ciuculescu MF, Daley H, MacKinnon B, Morris E, Federico A, Abriss D, Boardman K, Khelladi R, Shaw K, Negre H, Negre O, Nikiforow S, Ritz J, Pai SY, London WB, Dansereau C, Heeney MM, ...
The atlas of RNase H antisense oligonucleotide distribution and activity in the CNS of rodents and non-human primates following central administration
Jafar-Nejad P, Powers B, Soriano A, Zhao H, Norris DA, Matson J, DeBrosse-Serra B, Watson J, Narayanan P, Chun SJ, Mazur C, Kordasiewicz H, Swayze EE, Rigo F. Nucleic Acids Res. 2020 Dec 24:gkaa1235. Pubmed
Long-term safety and efficacy of patisiran for hereditary transthyretin-mediated amyloidosis with polyneuropathy: 12-month results of an open-label extension study
Adams D, Polydefkis M, González-Duarte A, Wixner J, Kristen AV, Schmidt HH, Berk JL, Losada López IA, Dispenzieri A, Quan D, Conceição IM, Slama MS, Gillmore JD, Kyriakides T, Ajroud-Driss S, Waddington-Cruz M, Mezei MM, Planté-Bordeneuve V, Attarian S, Mauricio E, Brannagan TH 3rd, Ueda ...
Pharmacokinetic and Pharmacodynamic Properties of Cemdisiran, an RNAi Therapeutic Targeting Complement Component 5, in Healthy Subjects and Patients with Paroxysmal Nocturnal Hemoglobinuria
Badri P, Jiang X, Borodovsky A, Najafian N, Kim J, Clausen VA, Goel V, Habtemariam B, Robbie GJ. Clin Pharmacokinet. 2020 Oct 13.
Safety and Immunogenicity of SARS-CoV-2 mRNA-1273 Vaccine in Older Adults
Anderson EJ, Rouphael NG, Widge AT, Jackson LA, Roberts PC, Makhene M, Chappell JD, Denison MR, Stevens LJ, Pruijssers AJ, McDermott AB, Flach B, Lin BC, Doria-Rose NA, O'Dell S, Schmidt SD, Corbett KS, Swanson PA 2nd, Padilla M, Neuzil KM, Bennett H, Leav B, ...
QR-313, an antisense oligonucleotide, shows therapeutic efficacy for treatment of dominant and recessive dystrophic epidermolysis bullosa: a preclinical study
Bornert O, Hogervorst M, Nauroy P, Bischof J, Swildens J, Athanasiou I, Tufa SF, Keene DR, Kiritsi D, Hainzl S, Murauer EM, Marinkovich MP, Platenburg G, Hausser I, Wally V, Ritsema T, Koller U, Haisma EM, Nyström A. J Invest Dermatol. 2020 Sep 15:S0022-202X(20)32061-3. Pubmed ...
