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Current Opinion in Genetics & Development|July 6, 2020
Nucleic acid therapeutics in neurodevelopmental diseaseAudrey M Winkelsas, Kenneth H FischbeckACS Chemical Biology|April 21, 2016
Inforna 2.0: A Platform for the Sequence-Based Design of Small Molecules Targeting Structured RNAsMatthew D Disney, Audrey M Winkelsas, Sai Pradeep Velagapudi, et al.Neurobiology of Disease|July 30, 2022
Altered SYNJ2BP-mediated mitochondrial-ER contacts in motor neuron diseaseNaemeh Pourshafie, Ester Masati, Amber Lopez, et al.Molecular Therapy. Nucleic Acids|February 12, 2021
Targeting the 5' untranslated region of SMN2 as a therapeutic strategy for spinal muscular atrophyAudrey M Winkelsas, Christopher Grunseich, George G Harmison, et al.Medrxiv : the Preprint Server for Health Sciences|July 2, 2026
A class of deep intronic IGHMBP2 variants activate a shared cryptic splice donor, enabling correction of select variants with a single antisense oligonucleotideSarah Silverstein, Andrew D Nguyen, Rotem Orbach, et al.Pageof 1