Design of Exon Skipping Oligonucleotides
Presenter: Annemieke Aartsma-Rus, PhD, Leiden University Medical Center Date: 20 February 2018 Description: Antisense-mediated splicing modulation utilizes antisense oligonucleotides (ASOs) to manipulate the splicing by sterically blocking the binding of splicing factors to pre-mRNA transcripts. While in industry often oligo walks are used to ...
Nucleic acid binding proteins affect the subcellular distribution of phosphorothioate antisense oligonucleotides
Bailey JK, Shen W, Liang XH, Crooke ST. Nucleic Acids Res. 2017 Oct 13;45(18):10649-10671. Dr. Jeffrey Bailey This paper is the latest in a series of publications from Stanley Crooke’s lab at Ionis investigating the interactions between phosphorothioate antisense oligonucleotides and intracellular proteins. ...
Interview with Vadim Y Dudkin, PhD
I first became exposed to oligonucleotide delivery while at Merck upon the acquisition of Sirna in 2006. Part of my work at the time involved glyco
Interview with Hannah Pendergraff, PhD
I became interested in oligo therapeutics when I started working in David Corey’s lab. I joined his group in 2009...
Interview with Keith T. Gagnon, PhD
I was thrown into the world of nucleic acid therapeutics during my postdoc with David Corey. Â My Ph.D. training focused on the...
Interview with Paul Burke, PhD
Paul Burke, PhD Principal, Burke Bioventures LLC How did you become interested in the field of oligonucleotides? I first became interested in
Interview with Kotaro Yoshioka, MD, PhD
Kotaro Yoshioka, MD, PhD Department of Neurology and Neurological Science, Tokyo Medical and Dental University How did you become interested in the field of
2017 Paper of the Year Award Winner: Richard Finkel, MD
Richard Finkel, MD Nemours Children’s Hospital Talk Title: Treatment of Infantile-onset Spinal Muscular Atrophy with Nusinersen: a Phase 2, Open-label, Dose-escalation Study Biography: Richard S. Finkel, MD is Professor of Neurology at the University of Central Florida School of Medicine, and Chief, Division of Neurology ...
2017 Gewirtz Memorial Scholarship Winner: Karyn Schmidt, PhD
Karyn Schmidt, PhDDana-Farber Cancer InstituteTalk Title: Inhibiting the Androgen Receptor Interaction with the Long Non-Coding RNA SLNCR using 2’-FANA-Modified Oligonucleotides Decreases Melanoma Invasion and Proliferation.Biography: Karyn Schmidt received her PhD in Biochemistry from the University of Rochester in 2013, where she studied in the laboratory of J. ...
Interview with Thorleif Møller, PhD
ThorleifMøller, PhD Mirrx Therapeutics A/S CEO & Founder How did you become interested in the field of oligonucleotides? During my PhD studies at the
