Newly Approved Drug Shows Clinical and Quality-of-Life Improvements for HAE Patients

October 15th, 2025|Categories: Featured Perspectives On Current Science|

Emily Wheeler was 19 when she noticed a gradual swelling in her left foot. Believing it was an allergic reaction, she went to her doctor, who told her it was gout and gave her several pills. An hour later, she started vomiting uncontrollably and ...

BLADE Directed Evolution of SaCas9 gRNAs Improves Gene Editing Efficiency

October 14th, 2025|Categories: Upcoming Webinars|

Date: December 11, 2025Time: 11-12pm EST / 5-6pm CET Register Here Title: BLADE Directed Evolution of SaCas9 gRNAs Improves Gene Editing Efficiency Description: CRISPR-Cas9 efficiency is often limited by misfolding between the sgRNA scaffold and spacer regions. Using the BLADE-SELEX directed evolution platform, we ...

New Approaches for Functional Oligonucleotides: RNA Activation and Non-Nucleosidic Aptamers.

October 14th, 2025|Categories: Upcoming Webinars|

Date: November 20, 2025Time: 11-12pm EST / 5-6pm CET Register Here Title: New Approaches for Functional Oligonucleotides: RNA Activation and Non-Nucleosidic Aptamers Description: This talk will highlight two complementary approaches aimed at expanding the functional scope of oligonucleotide therapeutics. First, I will present ...

DNA-based tools for the study and treatment of Parkinson’s Disease

October 14th, 2025|Categories: Upcoming Webinars|

Date: November 6, 2025 Time: 11-12pm EST / 5-6pm CET Register Here Title: DNA-based tools for the study and treatment of Parkinson’s DiseaseDescription:Aptamers are short stretches of DNA that fold into 3-dimensional shapes that can act as pockets for binding other biological molecules. This ...

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: Late Discovery Featured Paper of the Year|

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: Basic Featured Paper of the Year|

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 ...

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