α-Synuclein antisense oligonucleotides as a disease-modifying therapy for Parkinson’s disease
Papers of interest to society members in the field of oligonucleotide therapeutics. Antisense, aptamers, lncRNA, siRNA, splice modulation and ribozymes.
Antisense oligonucleotide therapy in a humanized mouse model of MECP2 duplication syndrome
Papers of interest to society members in the field of oligonucleotide therapeutics. Antisense, aptamers, lncRNA, siRNA, splice modulation and ribozymes.
Phase III Trial of ASO Therapy for the Treatment of Huntington’s Disease Halted
Recently, people who have been impacted by Huntington’s disease, their families, and the OTS community were disappointed to learn that the GENERATION HD1 Phase III study of Tominersen was halted based on the recommendation of an unblinded Independent Data Monitoring Committee. Although not based on [...]
Interview with Mark Behlke
Mark Behlke, MD, PhDChief Scientific OfficerIntegrated DNA Technologies, Inc. How did you become interested in the field of oligonucleotides?I had always thought that I would pursue a career in academic medicine, but in the Fall of 1995, I was visiting my parents who had moved to [...]
siRNA: From a Powerful Research Tool to Potent Therapeutic Modality
Earlier this year we celebrated the 20th Anniversary of RNA Interference in Mammalian Cells. In this article, we are going to discuss small interfering RNA (siRNA), a key mechanism that induces gene silencing in the RNAi pathway. siRNAs are highly specific, making them a [...]
The Astonishing History, Controversies, and Game-Changing Uses of CRISPR
Many of us view CRISPR as an incredibly useful tool. It is even referred to as genetic scissors because CRISPR allows scientists to edit the genes (the DNA) of organisms, from the smallest cells to an entire mammal. Because of its revolutionary impact on life [...]
Antisense Technology: A Review.
Papers of interest to society members in the field of oligonucleotide therapeutics. Antisense, aptamers, lncRNA, siRNA, splice modulation and ribozymes.
Best practices to ensure robust investigation of circular RNAs: pitfalls and tips
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
Sequence-specific 2′-O-methoxyethyl antisense oligonucleotides activate human platelets through glycoprotein VI, triggering formation of platelet-leukocyte aggregates
Slingsby MHL, Vijey P, Tsai IT, Roweth H, Couldwell G, Wilkie AR, Gaus H, Goolsby JM, Okazaki R, Terkovich BE, Semple JW, Thon JN, Henry SP, Narayanan P, Italiano JE Jr. Haematologica. 2021 Feb 11. Pubmed