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Bio
Defne Amado is a physician-scientist and Assistant Professor of Neurology at the University of Pennsylvania. Dr. Amado specializes in the development of gene therapy-based therapeutics for amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), and spinocerebellar ataxia (SCA). Her lab is particularly interested in using advanced gene editing and gene modulation tools, coupled with novel CNS-targeting delivery methods including evolved AAV capsids, to uncover novel therapeutic targets in ALS, FTD, and SCA pathogenesis using mouse and cell models of disease.
Dr. Amado earned her BA at Columbia University with a degree in Neuroscience, followed by an MD/PhD in Neuroscience at the University of Pennsylvania. She completed her thesis work in gene therapy for inherited retinal disease in the laboratory of Dr. Jean Bennett, where she worked on the development of Luxturna, the first FDA-approved gene therapy for an inherited disease. At Penn she also completed her Internship and Neurology residency followed by a Neuromuscular Research Fellowship supported by an NIH R25 and a subsequent Brody Family Medical Trust Fund Fellowship. Her first postdoctoral position was in the laboratory of Dr. Alice Chen-Plotkin, where she worked on identifying blood-based biomarkers of progression in neurodegenerative diseases and investigated a gene delivery-based approach to treatment of progranulin-deficient frontotemporal dementia. Her second postdoctoral position was in the laboratory of Dr. Beverly Davidson in which she used gene knockdown approaches to treat gain-of-function neurodegenerative diseases in animal and cell culture models. She is currently a K08-funded Assistant Professor of Neurology. Nonclinical interests include mentoring and scientific outreach, Sudoku, jigsaw puzzles, reading, and trying to beat her children in Battleship.
Research Interests
Papers共 40 篇Author StatisticsCo-AuthorSimilar Experts
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BIOLOGY-BASELno. 2 (2024): S250-S250
medRxiv : the preprint server for health sciences (2024)
medrxiv(2024)
Aaron Baldwin, Juliette Copeland, Meron Azage,Laynie Dratch,Kelsey Johnson,Rachel A Paul,Defne A Amado,Michael Baer,Andres Deik,Lauren B Elman,Michael Guo,Ali G Hamedani,
Defne A. Amado,Ashley B. Robbins,Alicia R. Smith, Katherine R. Whiteman, Guillem Chillon Bosch,Yonghong Chen, Joshua A. Fuller,Aleksandar Izda,Shareen Nelson, Abigail I. Dichter,Alex Mas Monteys,Beverly L. Davidson
bioRxiv the preprint server for biology (2024)
medRxiv : the preprint server for health sciences (2023)
Kelsey Johnson, Meron Azage, Nareen Babaian,Aaron Baldwin,Laynie Dratch,Rachel Paul,Tanya Bardakjian,Defne Amado, Pedro Gonzalez Alegre,Colin Quinn,Lauren Elman,Ali Hamedani,
ANNALS OF NEUROLOGY (2023): S215-S216
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Nature Communicationsno. 1 (2023): 1-20
Amyotrophic lateral sclerosis & frontotemporal degenerationno. 3-4 (2023): 317-325
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