Binding and Ligand Activation Driven Enrichment–Directed Evolution of SaCas9 gRNAs Improves Gene Editing Efficiency

October 2nd, 2025|Categories: Open Access NAT, Perspectives on Current Science|

Authors: Telmo Llanga, Korie Bush, Ying Sun, Amy Yan, Jonathan Zhou, Jan Gorodkin, and Bruce A. Sullenger AbstractClustered regularly interspaced short palindromic repeats-based editing is inefficient at over two-thirds of genetic targets. A primary cause is ribonucleic acid (RNA) misfolding that can occur between the ...

2025 Paper of the Year Award – Late Discovery Category

September 10th, 2025|Categories: Private|

Michele Carrer, Ph.D. Executive Director, Department of Neurology Ionis Pharmaceuticals, Carlsbad, CA “Conjugation to a transferrin receptor 1-binding Bicycle peptide enhances ASO and siRNA potency in skeletal and cardiac muscles” https://doi.org/10.1093/nar/gkaf270 Biography: Michele Carrer, Ph.D., is an Executive Director in the Department of Neurology ...

2025 Paper of the Year – Basic Research Category

September 10th, 2025|Categories: Private|

Ken Yamada, Ph.D. RTI, UMass Chan Medical School “Extended Nucleic Acid (exNA): A Novel Platform Technology Development for Enhancing siRNA Potency in Vivo” Paper of the Year: Enhancing siRNA efficacy in vivo with extended nucleic acid backbones https://doi.org/10.1038/s41587-024-02336-7 Biography: Dr. Ken Yamada is an ...

2025 Patient Advocacy Award

September 10th, 2025|Categories: Private|

Daniel Knight, PhD Candidate Carleton University “Strengthening Research, Advocacy, and Collaboration in ALS: The Dual Perspective of a Scientist and Community Member” Biography Daniel Knight is a fourth-year PhD candidate in chemistry at Carleton University in Ottawa, Canada, where he is developing a novel ...

2025 Dr. Alan M. Gewirtz Memorial Scholarship for Graduate Students

September 10th, 2025|Categories: Private|

Cristina Hofman, PhD Candidate UT Southwestern Medical Center “Nuclear Argonaute:miRNA complexes recognize target sequences within chromatin-associated RNA and silence gene expression” Biography: Cristina received her B.S. in Biochemistry and Molecular Biology at Trinity University and joined David Corey’s lab at the University of Texas ...

2025 Mary Ann Liebert Publishers Inc., Young Investigator Award

September 10th, 2025|Categories: Private|

Hien Tran Zhao, Ph.D. Ionis Pharmaceuticals, Inc. “Harnessing antisense oligonucleotide for the treatment of neurological disorders” Biography: Dr. Zhao has over 15 years of experience in Neuroscience Drug Discovery. She is currently an Executive Director of Neurology Research at Ionis Pharmaceuticals, Inc., a company ...

2025 OTS Lifetime Achievement Award Winner – Prof. Wojtek Stec

September 10th, 2025|Categories: Private|

Prof. Wojtek Stec (15 October 1940 - 4 December 2024) Biography: An outstanding chemist and a co-founder of Polish phosphorus chemistry of bioorganic compounds, Professor Stec was a distinguished scientific leader and a dedicated mentor. His research interests centered on bioorganic chemistry, with a ...

2025 OTS Lifetime Achievement Award Winner – Brett Monia, PhD

September 10th, 2025|Categories: Private|

Brett Monia, Ph.D. Chief Executive Officer, Ionis Pharmaceuticals "Pioneering RNA-Targeted Oligonucleotide Therapeutics: A 35-Year Journey" Biography: Dr. Monia is the chief executive officer, a member of the board of directors and a founding scientist of Ionis Pharmaceuticals. His contributions at Ionis include research into ...

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