Defne Amado

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Assistant Professor

University of Pennsylvania

United States

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About

Defne Amado is an Assistant Professor at the University of Pennsylvania in the United States. Her research interests include gene therapy for amyotrophic lateral sclerosis (ALS), digital markers of motor speech impairments, and disparities in genetic testing for neurologic disorders. Recent publications highlight her work on RNAi delivery systems, the importance of genetic sequencing in neuromuscular clinics, and the pathological mechanisms in ALS related to TDP-43 and SYNGAP1.

Recent Grants

Grant: Close

Viral-based Therapeutic Approaches for Reversal of ALS Pathology

Open Date: 2020-09-15

Close Date: 2025-06-30

Grant: Close

AAV-based strategies for treatment of amyotrophic lateral sclerosis

Open Date: 2018-07-01

Close Date: 2020-06-01

Grant: Close

AAV-mediated gene therapy for progranulin-deficient frontotemporal dementia

Open Date: 2016-07-01

Close Date: 2018-06-01

Grant: Close

Finding the diamonds in the coal mine: Using an unbiased screening approach to identify and

Open Date: 2014-07-01

Close Date: 2016-08-01

Grant: Close

Rod-dependent Cone Viability Factor: Towards a prevention of blindness

Open Date: 2007-06-01

Close Date: 2010-05-31

Articles (9)

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2025

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2024

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2024

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2024

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2023

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2022

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2021

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University of Pennsylvania

UNITED STATES

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Defne Amado

Research Interests

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Contact this professor

About

Recent Grants

Articles (9)

AAV-based delivery of RNAi targeting ataxin-2 improves survival and pathology in TDP-43 mice

Digital markers of motor speech impairments in spontaneous speech of patients with ALS-FTD spectrum disorders

Disparities in Genetic Testing for Neurologic Disorders

The Importance of Offering Exome or Genome Sequencing in Adult Neuromuscular Clinics

Mapping PTBP2 binding in human brain identifies SYNGAP1 as a target for therapeutic splice switching

Elevated Plasma Phosphorylated Tau 181 in Amyotrophic Lateral Sclerosis.

TMEM106B modifies TDP-43 pathology in human ALS brain and cell-based models of TDP-43 proteinopathy.

Gene therapy for ALS: A review

Multiple Streptococcal Brain Abscesses in a Patient Taking Secukinumab

Collaborators (7)

Melissa Asmar

DEBORAH FRENCH

Sunghye Cho

David Irwin

Mark Liberman

Sílvia Porta

Katheryn Alexandra Quilico Cousins

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