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Medchemcomm|December 14, 2018
Novel tricyclic glycal-based TRIB1 inducers that reprogram LDL metabolism in hepatic cellsMarek M Nagiec, Jeremy R Duvall, Adam P Skepner, et al.Scientific Reports|January 18, 2023
Molecular diagnosis and novel genes and phenotypes in a pediatric thoracic insufficiency cohortAlanna Strong, Meckenzie Behr, Carina Lott, et al.American Journal of Medical Genetics. Part A|October 16, 2021
A novel MBTPS2 variant associated with BRESHECK syndrome impairs sterol-regulated transcription and the endoplasmic reticulum stress responseAlanna Strong, Michael E March, Christopher J Cardinale, et al.Circulation. Genomic and Precision Medicine|April 15, 2018
Functional Assays to Screen and Dissect Genomic Hits: Doubling Down on the National Investment in Genomic ResearchKiran Musunuru, Daniel Bernstein, F Sessions Cole, et al.Nature Communications|May 15, 2023
GalNAc-Lipid nanoparticles enable non-LDLR dependent hepatic delivery of a CRISPR base editing therapyLisa N Kasiewicz, Souvik Biswas, Aaron Beach, et al.Circulation. Genomic and Precision Medicine|May 31, 2018
Interdisciplinary Models for Research and Clinical Endeavors in Genomic Medicine: A Scientific Statement From the American Heart AssociationKiran Musunuru, Pankaj Arora, John P Cooke, et al.Science Translational Medicine|April 19, 2019
In utero gene editing for monogenic lung diseaseDeepthi Alapati, William J Zacharias, Heather A Hartman, et al.Nature Medicine|October 10, 2018
In utero CRISPR-mediated therapeutic editing of metabolic genesAvery C Rossidis, John D Stratigis, Alexandra C Chadwick, et al.American Journal of Human Genetics|April 2, 2026
Implications of the FDA's new plausible mechanism framework for the development of a personalized in vivo prime editing platformEmily R Feierman, Madelynn N Whittaker, Aidan Quigley, et al.American Journal of Medical Genetics. Part A|May 26, 2025
Truncating Variants in RREB1 Cause a Novel RASopathy Syndrome of Congenital Heart Disease, Genitourinary Malformations, and Developmental DelayAlanna Strong, Caoimhe McKenna, Karen Stals, et al.Pageof 22