Defne Audrey Amado, MD, PHD

Amado DA, Davidson BL Gene Therapy for ALS: A Review , Molecular Therapy, 29(12): 2021,3345-3358

Defne A. Amado, Jaclyn Durkin, Katherine R. Whiteman, Joshua A. Fuller, Nancy M. Bonini, Beverly L. Davidson, Sigrid C. Veasey, and Matthew S. Kayser Sleep disturbances induced by TDP-43 proteinopathy are rescued by ATXN2 knockdown in Drosophila and mouse models of sporadic ALS , American Neurological Association Annual Meeting, Orlando, FL: 2024

Defne A. Amado, Alejandro Mas Monteys, Alicia R. Smith, Katherine Whiteman, Guillem Chillon Bosch, Ashley Robbins, Aleksandar Izda, Shareen Nelson, Abigail I. Dichter, Beverly L. Davidson Improved Survival, Strength, and Neuroinflammation in a Mouse Model of Sporadic ALS after Novel AAV-Mediated Delivery of RNAi Targeting Atxn2. , American Neurological Association Annual Meeting, Philadelphia, PA: 2023

Defne A. Amado, Alejandro Mas Monteys, Katherine Whiteman, Alicia R. Smith, Guillem Chillon Bosch, Aleksandar Izda, Beverly L. Davidson Novel AAV-Capsid-Mediated Delivery of an RNAi Targeting Atxn2 Extends Survival and Improves Strength and Neuroinflammation in a Mouse Model of Sporadic ALS , American Society of Gene and Cell Therapy Annual Meeting, Los Angeles, CA : 2023

Amado DA, Rieders JM, Diatta F, Hernandez-Con P, Singer A, Mak JT, Zhang J, Lancaster E, Davidson BL, Chen-Plotkin AS. AAV-mediated progranulin delivery to a mouse model of progranulin deficiency causes T cell-mediated toxicity. , Molecular Therapy, 27(2): 2019,465-478

Amado DA, Robbins A. The Emerging Landscape of Targeted Therapeutics for Genetic Neuromuscular Disorders. , Practical Neurology: 2023

Dawicki-McKenna J, Felix A, Waxman E, Cheng C, Amado DA, Ranum PT, Bogush A, Dungan LV, Maguire JA, Gagne AL, Heller EA, French DL, Davidson BL, Prosser B. Mapping PTBP2 binding in human brain identifies SYNGAP1 as a target for therapeutic splice switching. , Nature Communications, 14(1): 2023,2628

Mao F, Robinson J, Unger T, Amado DA, Elman L, Grossman M, Weintraub D, Wolk D, Lee E, Van Deerlin V, Porta SSs, Lee V, Trojanowski JQ, Chen-Plotkin AS. TMEM106B modifies TDP-43 pathology in human ALS brain and cell-based models of TDP-43 proteinopathy. , Acta Neuropathologica, 142(4): 2021,629-642

Amado D, Mingozzi F, Hui D, Bennicelli JL, Wei Z, Chen Y, Bote E, Grant RL, Golden JA, Narfstrom K, Syed NA, Orlin SE, High KA, Maguire AM, Bennett J. Safety and efficacy of subretinal readministration of a viral vector in large animals to treat congenital blindness. , Science Translational Medicine, 2(21): 2010,21ra16