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Fen-Biao Gao, PhD

Professor, RNA Therapeutics Institute, Department of Neurology, & Governor Paul Cellucci Chair in Neuroscience Research

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Research Focus - Pathogenic Mechanisms and Therapeutic Development in FTD and ALS

Our lab employs a combination of molecular, cellular, genetic, and behavioral approaches to dissect pathogenic mechanisms of frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). Using Drosophila, mouse models, and patient-specific induced pluripotent stem cells (iPSCs), we aim to identify key underlying pathogenic pathways as potential targets for therapeutic interventions.

Representative Publications

  • Lee S, Jun YW, Linares GR, Butler B, Yuva-Adyemir Y, Moore J, Krishnan G, Ruiz-Juarez B, Santana M, Pons M, Silverman N, Weng Z, Ichida JK, Gao FB. Downregulation of Hsp90 and the antimicrobial peptide Mtk suppresses poly(GR)-induced neurotoxicity in C9ORF72-ALS/FTD. 2023 Mar 10:S0896-6273(23)00133-2. doi: 10.1016/j. Neuron. 2023.02.029. Read Publication
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In the News

  • New study shows nuclear RNA foci harmless in fruit fly model of C90RF72 ALS/FTD
    Oct 14, 2015

    New study shows nuclear RNA foci harmless in fruit fly model of C90RF72 ALS/FTD

    Abnormal protein production, not nuclear RNA foci, is a major source of toxicity in the most common genetic form of amyotrophic lateral sclerosis and frontotemporal dementia, according to a new study by Fen-Biao Gao, PhD, published in the journal Neuron.

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  • Fen-Biao Gao, inaugural Cellucci Chair, studies ALS, FTD
    Aug 1, 2017

    Fen-Biao Gao, inaugural Cellucci Chair, studies ALS, FTD

    Fen-Biao Gao, PhD, the Governor Paul Cellucci Chair in Neuroscience Research and professor of neurology, studies the genetic mutations that cause amyotrophic lateral sclerosis (ALS) and frontotemporal dementia.

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  • UMMS scientists find age-related DNA damage linked to repeat expansion in ALS and FTD
    Oct 7, 2016

    UMMS scientists find age-related DNA damage linked to repeat expansion in ALS and FTD

    Research by Fen-Biao Gao, PhD, shows that age-related DNA damage caused by oxidative stress is a key contributor to the breakdown of motor neurons in some amyotrophic lateral sclerosis and frontotemporal dementia patients.

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  • Potential drug targets for ALS and FTD identified in two Fen-Biao Gao studies
    May 14, 2019

    Potential drug targets for ALS and FTD identified in two Fen-Biao Gao studies

    A pair of collaborative studies led by Fen-Biao Gao, PhD, have identified two potential drug targets for the diseases amyotrophic lateral sclerosis and frontotemporal dementia. The studies provide a new layer of detail about how the most common genetic mutation responsible for both ALS and FTD causes neuron cell death.

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  • How frontotemporal dementia changes the brain
    Feb 23, 2023

    How frontotemporal dementia changes the brain

    In a piece written for The Conversation, Fen-Biao Gao, PhD, talks about how frontotemporal dementia changes the brain and the research that is untangling its genetic causes.

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  • Researchers reveal how a common mutation causes neurodegenerative disease
    Aug 27, 2015

    Researchers reveal how a common mutation causes neurodegenerative disease

    Fen-Biao Gao, PhD, and colleagues have determined how the most common gene mutation in amyotrophic lateral sclerosis and frontotemporal dementia disrupts normal cell function by interfering with the movement of RNAs and proteins into and out of the nucleus.

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  • Fen-Biao Gao receives Javits Neuroscience Investigator Award
    Jan 2, 2018

    Fen-Biao Gao receives Javits Neuroscience Investigator Award

    Fen-Biao Gao, PhD, was selected to receive a Javits Neuroscience Investigator Award, a conditional seven-year, $4.16 million grant from the National Institute of Neurological Disorders and Stroke.

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  • Fen-Biao Gao details how frontotemporal dementia changes the brain and explores its genetic causes
    Feb 26, 2024

    Fen-Biao Gao details how frontotemporal dementia changes the brain and explores its genetic causes

    In an updated article originally published by The Conversation in Feb. 2023, Fen-Biao Gao, PhD, talks about how frontotemporal dementia changes the brain and the research that is untangling its genetic causes.

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