The paper enhances Nme2Cas9 and Nme2SmuCas9 nucleases and base editors for improved efficiency and vector compatibility through mutagenesis and deaminase linker optimization.

bioRxiv · Oct 30, 2024 · PreprintMolecular Biology

Interface-guided phenotyping of coding variants in the transcription factor RUNX1 with SEUSS

Kıvılcım Öztürk, Rebecca Panwala, Jeanna Sheen, Kyle M. Ford, Nathan Payne, Dong‐Er Zhang, Stephan Hütter, Torsten Haferlach, Trey E. Ideker, Prashant Mali, Hannah K. Carter

The paper uses SEUSS to analyze the effects of 115 RUNX1 mutations on RNA profiles in myelogenous leukemia cells, categorizing them into three functional groups.

bioRxiv · Aug 3, 2023 · PreprintBioinformatics

Self-delivering CRISPR RNAs for AAV Co-delivery and Genome Editing in vivo

Han Zhang, Karen L. Kelly, Jonathan Lee, Dimas Echeverria, David Cooper, Rebecca Panwala, Zexiang Chen, Nicholas Gaston, Gregory A. Newby, Jun Xie, David R. Liu, Guangping Gao, Scot A. Wolfe, Anastasia Khvorova, Jonathan K. Watts, Erik J. Sontheimer

This study demonstrates the use of protecting oligos to enhance crRNA stability and potency for in vivo genome editing via AAV co-delivery in mice.

bioRxiv · Mar 20, 2023 · PreprintMolecular Biology