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Fen-Biao Gao PhD

Endowed TitleGovernor Paul Cellucci Chair in Neuroscience Research
InstitutionUMass Chan Medical School
AddressUMass Chan Medical School
368 Plantation Street Room AS6-1051
Worcester MA 01605
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    Other Positions
    InstitutionT.H. Chan School of Medicine

    InstitutionT.H. Chan School of Medicine
    DepartmentNeuroNexus Institute

    InstitutionT.H. Chan School of Medicine
    DepartmentRNA Therapeutics Institute

    InstitutionMorningside Graduate School of Biomedical Sciences
    DepartmentInterdisciplinary Graduate Program

    InstitutionMorningside Graduate School of Biomedical Sciences
    DepartmentMD/PhD Program

    InstitutionMorningside Graduate School of Biomedical Sciences

    InstitutionMorningside Graduate School of Biomedical Sciences
    DepartmentPostbaccalaureate Research Education Program

    InstitutionMorningside Graduate School of Biomedical Sciences
    DepartmentTranslational Science

    Collapse Biography 
    Collapse education and training
    Peking University, Beijing, , ChinaBSBiochemistry
    Tsinghua University, Beijing, , ChinaMSBiochemistry
    Duke University, Durham, NC, United StatesPHDMolecular Biology
    University College London, London, UKPostdoc 1997Developmental Neurobiology
    University of California, San Francisco (UCSF)Postdoc2000Developmental Neurobiology
    Collapse awards and honors
    1995Departmental Norman Conant Award, Duke University
    1995 - 1998Hitchings-Elion Fellowship, Burroughs Wellcome Fund
    1998 - 2000Senior Postdoctoral Fellowship, American Cancer Society California Chapter
    2001Sloan Research Fellow in Neuroscience, Alfred P. Sloan Foundation
    2002Klingenstein Fellow in Neuroscience, Esther A. and Joseph Klingenstein Fund
    2002McKnight Neuroscience of Brain Disorders Award, McKnight Foundation
    2017Governor Paul Cellucci Chair in Neuroscience Research, UMMS
    2017Javits Neuroscience Investigator Award, NIH

    Collapse Overview 
    Collapse Summary
    Our lab uses a combination of molecular, cellular, genetic, and behavioral approaches to further dissect the pathogenic mechanisms of Frontotemporal Dementia and Amyotrophic Lateral Sclerosis with a focus on mutant CHMP2B, progranulin and C9ORF72. We will identify common underlying pathways as potential targets for therapeutic interventions.
    Collapse overview

     Research Focus: Understanding ALS and Frontotemporal Dementia 

    Dr. Fen-Biao GaoFen-Biao Gao Lab Website

    Frontotemporal dementia (FTD) is an age-dependent neurodegenerative condition associated with focal atrophy of the frontal and/or temporal lobes and recognized now as the most common form of dementia before the age of 60. Unfortunately, the molecular pathogenesis of FTD remains largely unknown and effective treatments are not available. Recent exciting advances indicate that FTD is often associated with amyotrophic lateral sclerosis (ALS) and several genes are involved in thepathogenesis of both ALS and FTD, including CHMP2B, TDP-43, FUS, TBK1, and C9ORF72. How these mutant proteins cause or contribute to neuronal dysfunction and neurodegeneration in ALS and FTD remains poorly defined.

    A few years ago, we cloned anovel Drosophila gene called shrub, which encodes a key component of ESCRT-III and regulates dendritic morphogenesis. In cultured cortical neurons, we found that dysfunctional ESCRT-III, lacking essential components (such as mSnf7-2, one of the mouse homologs of Shrub) or containing ectopically expressed FTD3-associated mutant CHMP2B, causes dendritic retraction,autophagosome accumulation and eventual neurodegeneration. Through an unbiased genetic screen in a Drosophila modelof FTD3, we identified several genetic modifiers of CHMP2B toxicity in vivo that are currently being characterized. We have also been using Drosophila models to study other ALS/FTD genes, in paticular, C9ORF72.

    We have also established patient-specific induced pluripotent stem cells (iPSC) models of FTD and ALS with mutations in progranulin, TDP-43, C9ORF72 and other genes. Over the next a few years, we will use a combination of molecular, cellular, and genetic approaches in both Drosophila and iPSCs models to further dissect the pathogenic mechanisms involving these ALS/FTD disease genes. Our ultimate goal is to identify common underlying pathogenic pathways as potential targets for therapeutic interventions in both ALS and FTD.

    Collapse Rotation Projects

    Project 1: Perform genetic and molecular analysis of disease modifiers identified from our fly models of ALS and FTD. 

    Project 2: Study pathogenic mechanisms of ALS/FTD in patient-specific iPSC derived cortical and motor neurons.

    Collapse Bibliographic 
    Collapse selected publications
    Publications listed below are automatically derived from MEDLINE/PubMed and other sources, which might result in incorrect or missing publications. Faculty can login to make corrections and additions.
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    PMC Citations indicate the number of times the publication was cited by articles in PubMed Central, and the Altmetric score represents citations in news articles and social media. (Note that publications are often cited in additional ways that are not shown here.) Fields are based on how the National Library of Medicine (NLM) classifies the publication's journal and might not represent the specific topic of the publication. Translation tags are based on the publication type and the MeSH terms NLM assigns to the publication. Some publications (especially newer ones and publications not in PubMed) might not yet be assigned Field or Translation tags.) Click a Field or Translation tag to filter the publications.
    1. Halim DO, Munson M, Gao FB. The exocyst complex in neurological disorders. Hum Genet. 2023 Apr 22. PMID: 37085629.
      Citations:    Fields:    
    2. 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. Neuron. 2023 05 03; 111(9):1381-1390.e6. PMID: 36931278.
      Citations:    Fields:    
    3. Jun YW, Lee S, Ban BK, Lee JA, Gao FB. Non-muscle MYH10/myosin IIB recruits ESCRT-III to participate in autophagosome closure to maintain neuronal homeostasis. Autophagy. 2023 Feb 27; 1-17. PMID: 36849436.
      Citations:    Fields:    
    4. Chen Y, Krishnan G, Parsi S, Pons M, Nikolaki V, Cao L, Xu Z, Gao FB. The enhanced association between mutant CHMP2B and spastin is a novel pathological link between frontotemporal dementia and hereditary spastic paraplegias. Acta Neuropathol Commun. 2022 11 22; 10(1):169. PMID: 36414997.
      Citations:    Fields:    Translation:HumansAnimals
    5. Meijboom KE, Abdallah A, Fordham NP, Nagase H, Rodriguez T, Kraus C, Gendron TF, Krishnan G, Esanov R, Andrade NS, Rybin MJ, Ramic M, Stephens ZD, Edraki A, Blackwood MT, Kahriman A, Henninger N, Kocher JA, Benatar M, Brodsky MH, Petrucelli L, Gao FB, Sontheimer EJ, Brown RH, Zeier Z, Mueller C. CRISPR/Cas9-mediated excision of ALS/FTD-causing hexanucleotide repeat expansion in C9ORF72 rescues major disease mechanisms in vivo and in vitro. Nat Commun. 2022 10 21; 13(1):6286. PMID: 36271076.
      Citations: 2     Fields:    Translation:AnimalsCells
    6. Krishnan G, Raitcheva D, Bartlett D, Prudencio M, McKenna-Yasek DM, Douthwright C, Oskarsson BE, Ladha S, King OD, Barmada SJ, Miller TM, Bowser R, Watts JK, Petrucelli L, Brown RH, Kankel MW, Gao FB. Poly(GR) and poly(GA) in cerebrospinal fluid as potential biomarkers for C9ORF72-ALS/FTD. Nat Commun. 2022 05 19; 13(1):2799. PMID: 35589711.
      Citations: 7     Fields:    Translation:Humans
    7. Loveland AB, Svidritskiy E, Susorov D, Lee S, Park A, Zvornicanin S, Demo G, Gao FB, Korostelev AA. Ribosome inhibition by C9ORF72-ALS/FTD-associated poly-PR and poly-GR proteins revealed by cryo-EM. Nat Commun. 2022 05 19; 13(1):2776. PMID: 35589706.
      Citations: 8     Fields:    Translation:HumansCells
    8. Halim D, Gao FB. RNA targets of TDP-43: Which one is more important in neurodegeneration? Transl Neurodegener. 2022 02 25; 11(1):12. PMID: 35209956.
      Citations:    Fields:    
    9. van 't Spijker HM, Stackpole EE, Almeida S, Katsara O, Liu B, Shen K, Schneider RJ, Gao FB, Richter JD. Ribosome profiling reveals novel regulation of C9ORF72 GGGGCC repeat-containing RNA translation. RNA. 2022 02; 28(2):123-138. PMID: 34848561.
      Citations: 6     Fields:    Translation:HumansCells
    10. Lu Y, Almeida S, Gao FB. TBK1 haploinsufficiency in ALS and FTD compromises membrane trafficking. Acta Neuropathol. 2021 07; 142(1):217-221. PMID: 34081168.
      Citations: 2     Fields:    Translation:HumansCells
    11. Lampasona A, Almeida S, Gao FB. Translation of the poly(GR) frame in C9ORF72-ALS/FTD is regulated by cis-elements involved in alternative splicing. Neurobiol Aging. 2021 09; 105:327-332. PMID: 34157654.
      Citations: 2     Fields:    Translation:HumansAnimalsCells
    12. Maor-Nof M, Shipony Z, Lopez-Gonzalez R, Nakayama L, Zhang YJ, Couthouis J, Blum JA, Castruita PA, Linares GR, Ruan K, Ramaswami G, Simon DJ, Nof A, Santana M, Han K, Sinnott-Armstrong N, Bassik MC, Geschwind DH, Tessier-Lavigne M, Attardi LD, Lloyd TE, Ichida JK, Gao FB, Greenleaf WJ, Yokoyama JS, Petrucelli L, Gitler AD. p53 is a central regulator driving neurodegeneration caused by C9orf72 poly(PR). Cell. 2021 02 04; 184(3):689-708.e20. PMID: 33482083.
      Citations: 46     Fields:    Translation:AnimalsCells
    13. Lu Y, West RJH, Pons M, Sweeney ST, Gao FB. Ik2/TBK1 and Hook/Dynein, an adaptor complex for early endosome transport, are genetic modifiers of FTD-associated mutant CHMP2B toxicity in Drosophila. Sci Rep. 2020 08 26; 10(1):14221. PMID: 32848189.
      Citations: 4     Fields:    Translation:AnimalsCells
    14. Li S, Wu Z, Li Y, Tantray I, De Stefani D, Mattarei A, Krishnan G, Gao FB, Vogel H, Lu B. Altered MICOS Morphology and Mitochondrial Ion Homeostasis Contribute to Poly(GR) Toxicity Associated with C9-ALS/FTD. Cell Rep. 2020 08 04; 32(5):107989. PMID: 32755582.
      Citations: 14     Fields:    Translation:HumansAnimalsCells
    15. Krishnan G, Zhang Y, Gu Y, Kankel MW, Gao FB, Almeida S. CRISPR deletion of the C9ORF72 promoter in ALS/FTD patient motor neurons abolishes production of dipeptide repeat proteins and rescues neurodegeneration. Acta Neuropathol. 2020 07; 140(1):81-84. PMID: 32266467.
      Citations: 13     Fields:    Translation:HumansCells
    16. Yuva-Aydemir Y, Almeida S, Krishnan G, Gendron TF, Gao FB. Transcription elongation factor AFF2/FMR2 regulates expression of expanded GGGGCC repeat-containing C9ORF72 allele in ALS/FTD. Nat Commun. 2019 11 29; 10(1):5466. PMID: 31784536.
      Citations: 24     Fields:    Translation:HumansAnimalsCells
    17. Almeida S, Krishnan G, Rushe M, Gu Y, Kankel MW, Gao FB. Production of poly(GA) in C9ORF72 patient motor neurons derived from induced pluripotent stem cells. Acta Neuropathol. 2019 12; 138(6):1099-1101. PMID: 31624870.
      Citations: 13     Fields:    Translation:HumansCells
    18. Cheng W, Wang S, Zhang Z, Morgens DW, Hayes LR, Lee S, Portz B, Xie Y, Nguyen BV, Haney MS, Yan S, Dong D, Coyne AN, Yang J, Xian F, Cleveland DW, Qiu Z, Rothstein JD, Shorter J, Gao FB, Bassik MC, Sun S. CRISPR-Cas9 Screens Identify the RNA Helicase DDX3X as a Repressor of C9ORF72 (GGGGCC)n Repeat-Associated Non-AUG Translation. Neuron. 2019 12 04; 104(5):885-898.e8. PMID: 31587919.
      Citations: 52     Fields:    Translation:HumansAnimalsCells
    19. Choi SY, Lopez-Gonzalez R, Krishnan G, Phillips HL, Li AN, Seeley WW, Yao WD, Almeida S, Gao FB. C9ORF72-ALS/FTD-associated poly(GR) binds Atp5a1 and compromises mitochondrial function in vivo. Nat Neurosci. 2019 06; 22(6):851-862. PMID: 31086314.
      Citations: 91     Fields:    Translation:HumansAnimalsCells
    20. Lopez-Gonzalez R, Yang D, Pribadi M, Kim TS, Krishnan G, Choi SY, Lee S, Coppola G, Gao FB. Partial inhibition of the overactivated Ku80-dependent DNA repair pathway rescues neurodegeneration in C9ORF72-ALS/FTD. Proc Natl Acad Sci U S A. 2019 05 07; 116(19):9628-9633. PMID: 31019093.
      Citations: 39     Fields:    Translation:AnimalsCells
    21. Yuva-Aydemir Y, Almeida S, Gao FB. Insights into C9ORF72-Related ALS/FTD from Drosophila and iPSC Models. Trends Neurosci. 2018 07; 41(7):457-469. PMID: 29729808.
      Citations: 29     Fields:    Translation:HumansAnimalsCells
    22. West RJH, Ugbode C, Gao FB, Sweeney ST. The pro-apoptotic JNK scaffold POSH/SH3RF1 mediates CHMP2BIntron5-associated toxicity in animal models of frontotemporal dementia. Hum Mol Genet. 2018 04 15; 27(8):1382-1395. PMID: 29432529.
      Citations: 7     Fields:    Translation:HumansAnimalsCells
    23. Liu X, He J, Gao FB, Gitler AD, Fan D. The epidemiology and genetics of Amyotrophic lateral sclerosis in China. Brain Res. 2018 08 15; 1693(Pt A):121-126. PMID: 29501653.
      Citations: 18     Fields:    Translation:HumansCells
    24. Markmiller S, Soltanieh S, Server KL, Mak R, Jin W, Fang MY, Luo EC, Krach F, Yang D, Sen A, Fulzele A, Wozniak JM, Gonzalez DJ, Kankel MW, Gao FB, Bennett EJ, L?cuyer E, Yeo GW. Context-Dependent and Disease-Specific Diversity in Protein Interactions within Stress Granules. Cell. 2018 01 25; 172(3):590-604.e13. PMID: 29373831.
      Citations: 301     Fields:    Translation:HumansAnimalsCells
    25. Gao FB, Richter JD, Cleveland DW. Rethinking Unconventional Translation in Neurodegeneration. Cell. 2017 Nov 16; 171(5):994-1000. PMID: 29149615.
      Citations: 33     Fields:    Translation:HumansAnimalsCells
    26. Gao FB, Almeida S, Lopez-Gonzalez R. Dysregulated molecular pathways in amyotrophic lateral sclerosis-frontotemporal dementia spectrum disorder. EMBO J. 2017 10 16; 36(20):2931-2950. PMID: 28916614.
      Citations: 87     Fields:    Translation:HumansAnimals
    27. Lee CW, Stankowski JN, Chew J, Cook CN, Lam YW, Almeida S, Carlomagno Y, Lau KF, Prudencio M, Gao FB, Bogyo M, Dickson DW, Petrucelli L. The lysosomal protein cathepsin L is a progranulin protease. Mol Neurodegener. 2017 07 25; 12(1):55. PMID: 28743268.
      Citations: 45     Fields:    Translation:HumansCells
    28. Yin S, Lopez-Gonzalez R, Kunz RC, Gangopadhyay J, Borufka C, Gygi SP, Gao FB, Reed R. Evidence that C9ORF72 Dipeptide Repeat Proteins Associate with U2 snRNP to Cause Mis-splicing in ALS/FTD Patients. Cell Rep. 2017 06 13; 19(11):2244-2256. PMID: 28614712.
      Citations: 55     Fields:    Translation:HumansCells
    29. Wu CH, Giampetruzzi A, Tran H, Fallini C, Gao FB, Landers JE. A Drosophila model of ALS reveals a partial loss of function of causative human PFN1 mutants. Hum Mol Genet. 2017 06 01; 26(11):2146-2155. PMID: 28379367.
      Citations: 8     Fields:    Translation:HumansAnimalsCells
    30. Gao FB, Richter JD. Microsatellite Expansion Diseases: Repeat Toxicity Found in Translation. Neuron. 2017 Jan 18; 93(2):249-251. PMID: 28103472.
      Citations: 6     Fields:    Translation:HumansCells
    31. Lee JA, Yue Z, Gao FB. Autophagy in neurodegenerative diseases. Brain Res. 2016 10 15; 1649(Pt B):141-142. PMID: 27712723.
      Citations: 4     Fields:    Translation:HumansAnimals
    32. Lopez-Gonzalez R, Lu Y, Gendron TF, Karydas A, Tran H, Yang D, Petrucelli L, Miller BL, Almeida S, Gao FB. Poly(GR) in C9ORF72-Related ALS/FTD Compromises Mitochondrial Function and Increases Oxidative Stress and DNA Damage in iPSC-Derived Motor Neurons. Neuron. 2016 Oct 19; 92(2):383-391. PMID: 27720481.
      Citations: 187     Fields:    Translation:HumansCells
    33. Silva MC, Cheng C, Mair W, Almeida S, Fong H, Biswas MHU, Zhang Z, Huang Y, Temple S, Coppola G, Geschwind DH, Karydas A, Miller BL, Kosik KS, Gao FB, Steen JA, Haggarty SJ. Human iPSC-Derived Neuronal Model of Tau-A152T Frontotemporal Dementia Reveals Tau-Mediated Mechanisms of Neuronal Vulnerability. Stem Cell Reports. 2016 09 13; 7(3):325-340. PMID: 27594585.
      Citations: 44     Fields:    Translation:HumansCells
    34. Biswas MHU, Almeida S, Lopez-Gonzalez R, Mao W, Zhang Z, Karydas A, Geschwind MD, Biernat J, Mandelkow EM, Futai K, Miller BL, Gao FB. MMP-9 and MMP-2 Contribute to Neuronal Cell Death in iPSC Models of Frontotemporal Dementia with MAPT Mutations. Stem Cell Reports. 2016 09 13; 7(3):316-324. PMID: 27594586.
      Citations: 14     Fields:    Translation:HumansCells
    35. Kramer NJ, Carlomagno Y, Zhang YJ, Almeida S, Cook CN, Gendron TF, Prudencio M, Van Blitterswijk M, Belzil V, Couthouis J, Paul JW, Goodman LD, Daughrity L, Chew J, Garrett A, Pregent L, Jansen-West K, Tabassian LJ, Rademakers R, Boylan K, Graff-Radford NR, Josephs KA, Parisi JE, Knopman DS, Petersen RC, Boeve BF, Deng N, Feng Y, Cheng TH, Dickson DW, Cohen SN, Bonini NM, Link CD, Gao FB, Petrucelli L, Gitler AD. Spt4 selectively regulates the expression of C9orf72 sense and antisense mutant transcripts. Science. 2016 Aug 12; 353(6300):708-12. PMID: 27516603.
      Citations: 73     Fields:    Translation:HumansAnimalsCells
    36. Swartz EW, Baek J, Pribadi M, Wojta KJ, Almeida S, Karydas A, Gao FB, Miller BL, Coppola G. A Novel Protocol for Directed Differentiation of C9orf72-Associated Human Induced Pluripotent Stem Cells Into Contractile Skeletal Myotubes. Stem Cells Transl Med. 2016 Nov; 5(11):1461-1472. PMID: 27369896.
      Citations: 19     Fields:    
    37. Almeida S, Gao FB. Lost & found: C9ORF72 and the autophagy pathway in ALS/FTD. EMBO J. 2016 06 15; 35(12):1251-3. PMID: 27154207.
      Citations: 6     Fields:    Translation:Humans
    38. Almeida S, Gao F, Coppola G, Gao FB. Suberoylanilide hydroxamic acid increases progranulin production in iPSC-derived cortical neurons of frontotemporal dementia patients. Neurobiol Aging. 2016 06; 42:35-40. PMID: 27143419.
      Citations: 7     Fields:    Translation:HumansCells
    39. Woehlbier U, Colombo A, Saaranen MJ, P?rez V, Ojeda J, Bustos FJ, Andreu CI, Torres M, Valenzuela V, Medinas DB, Rozas P, Vidal RL, Lopez-Gonzalez R, Salameh J, Fernandez-Collemann S, Mu?oz N, Matus S, Armisen R, Sagredo A, Palma K, Irrazabal T, Almeida S, Gonzalez-Perez P, Campero M, Gao FB, Henny P, van Zundert B, Ruddock LW, Concha ML, Henriquez JP, Brown RH, Hetz C. ALS-linked protein disulfide isomerase variants cause motor dysfunction. EMBO J. 2016 Apr 15; 35(8):845-65. PMID: 26869642.
      Citations: 39     Fields:    Translation:HumansAnimalsCells
    40. Burguete AS, Almeida S, Gao FB, Kalb R, Akins MR, Bonini NM. GGGGCC microsatellite RNA is neuritically localized, induces branching defects, and perturbs transport granule function. Elife. 2015 Dec 09; 4:e08881. PMID: 26650351.
      Citations: 57     Fields:    Translation:HumansAnimalsCells
    41. Peters OM, Cabrera GT, Tran H, Gendron TF, McKeon JE, Metterville J, Weiss A, Wightman N, Salameh J, Kim J, Sun H, Boylan KB, Dickson D, Kennedy Z, Lin Z, Zhang YJ, Daughrity L, Jung C, Gao FB, Sapp PC, Horvitz HR, Bosco DA, Brown SP, de Jong P, Petrucelli L, Mueller C, Brown RH. Human C9ORF72 Hexanucleotide Expansion Reproduces RNA Foci and Dipeptide Repeat Proteins but Not Neurodegeneration in BAC Transgenic Mice. Neuron. 2015 Dec 02; 88(5):902-909. PMID: 26637797.
      Citations: 139     Fields:    Translation:HumansAnimalsCells
    42. Tran H, Almeida S, Moore J, Gendron TF, Chalasani U, Lu Y, Du X, Nickerson JA, Petrucelli L, Weng Z, Gao F-B. (2015). Differential toxicity of nuclear RNA foci versus dipeptide repeat proteins in a Drosophila model of C9ORF72 FTD/ALS. Neuron. 2015; (87):1207-1214.
    43. Freibaum B*, Lu Y*, Lopez-Gonzalez R, Kim NC, Almeida S, Lee K-H, Badders N, Valentine M, Miller BL, Wong PC, Petrucelli L, Kim HJ, Gao F-B,‡ Taylor JP.‡ (2015). GGGGCC repeat expansion in C9ORF72 compromises nucleocytoplasmic transport. Nature. 2015; 525(129-133):(*: co-first author; ‡: co-PI).
    44. Yang D, Abdallah A, Li Z, Lu Y, Almeida S, Gao FB. FTD/ALS-associated poly(GR) protein impairs the Notch pathway and is recruited by poly(GA) into cytoplasmic inclusions. Acta Neuropathol. 2015 Oct; 130(4):525-35. PMID: 26031661.
      Citations: 75     Fields:    Translation:HumansAnimalsCells
    45. Yuva-Aydemir Y, Xu XL, Aydemir O, Gascon E, Sayin S, Zhou W, Hong Y, Gao FB. Downregulation of the Host Gene jigr1 by miR-92 Is Essential for Neuroblast Self-Renewal in Drosophila. PLoS Genet. 2015 May; 11(5):e1005264. PMID: 26000445.
      Citations: 23     Fields:    Translation:AnimalsCells
    46. West RJ, Lu Y, Marie B, Gao FB, Sweeney ST. Rab8, POSH, and TAK1 regulate synaptic growth in a Drosophila model of frontotemporal dementia. J Cell Biol. 2015 Mar 30; 208(7):931-47. PMID: 25800055.
      Citations: 41     Fields:    Translation:AnimalsCells
    47. Gascon E, Lynch K, Ruan H, Almeida S, Verheyden JM, Seeley WW, Dickson DW, Petrucelli L, Sun D, Jiao J, Zhou H, Jakovcevski M, Akbarian S, Yao WD, Gao FB. Alterations in microRNA-124 and AMPA receptors contribute to social behavioral deficits in frontotemporal dementia. Nat Med. 2014 Dec; 20(12):1444-51. PMID: 25401692.
      Citations: 87     Fields:    Translation:AnimalsCells
    48. Gao FB, Taylor JP. RNA metabolism in neurological disease. Brain Res. 2014 Oct 10; 1584:1-2. PMID: 25248315.
      Citations: 1     Fields:    Translation:Humans
    49. Simkin AT, Bailey JA, Gao FB, Jensen JD. Inferring the evolutionary history of primate microRNA binding sites: overcoming motif counting biases. Mol Biol Evol. 2014 Jul; 31(7):1894-901. PMID: 24723422.
      Citations: 4     Fields:    Translation:HumansAnimalsCells
    50. Gascon E, Gao FB. The emerging roles of microRNAs in the pathogenesis of frontotemporal dementia-amyotrophic lateral sclerosis (FTD-ALS) spectrum disorders. J Neurogenet. 2014 Mar-Jun; 28(1-2):30-40. PMID: 24506814.
      Citations: 24     Fields:    Translation:Humans
    51. Lee WC, Almeida S, Prudencio M, Caulfield TR, Zhang YJ, Tay WM, Bauer PO, Chew J, Sasaguri H, Jansen-West KR, Gendron TF, Stetler CT, Finch N, Mackenzie IR, Rademakers R, Gao FB, Petrucelli L. Targeted manipulation of the sortilin-progranulin axis rescues progranulin haploinsufficiency. Hum Mol Genet. 2014 Mar 15; 23(6):1467-78. PMID: 24163244.
      Citations: 41     Fields:    Translation:HumansCells
    52. Cheruiyot A, Lee JA, Gao FB, Ahmad ST. Expression of mutant CHMP2B, an ESCRT-III component involved in frontotemporal dementia, causes eye deformities due to Notch misregulation in Drosophila. FASEB J. 2014 Feb; 28(2):667-75. PMID: 24158394.
      Citations: 6     Fields:    Translation:Animals
    53. Zhang Z, Almeida S, Lu Y, Nishimura AL, Peng L, Sun D, Wu B, Karydas AM, Tartaglia MC, Fong JC, Miller BL, Farese RV, Moore MJ, Shaw CE, Gao FB. Downregulation of microRNA-9 in iPSC-derived neurons of FTD/ALS patients with TDP-43 mutations. PLoS One. 2013; 8(10):e76055. PMID: 24143176.
      Citations: 67     Fields:    Translation:HumansCells
    54. Lu Y, Zhang Z, Sun D, Sweeney ST, Gao FB. Syntaxin 13, a genetic modifier of mutant CHMP2B in frontotemporal dementia, is required for autophagosome maturation. Mol Cell. 2013 Oct 24; 52(2):264-71. PMID: 24095276.
      Citations: 33     Fields:    Translation:HumansAnimalsCells
    55. Almeida S, Gascon E, Tran H, Chou HJ, Gendron TF, Degroot S, Tapper AR, Sellier C, Charlet-Berguerand N, Karydas A, Seeley WW, Boxer AL, Petrucelli L, Miller BL, Gao FB. Modeling key pathological features of frontotemporal dementia with C9ORF72 repeat expansion in iPSC-derived human neurons. Acta Neuropathol. 2013 Sep; 126(3):385-99. PMID: 23836290.
      Citations: 183     Fields:    Translation:HumansAnimalsCells
    56. Filiano AJ, Martens LH, Young AH, Warmus BA, Zhou P, Diaz-Ramirez G, Jiao J, Zhang Z, Huang EJ, Gao FB, Farese RV, Roberson ED. Dissociation of frontotemporal dementia-related deficits and neuroinflammation in progranulin haploinsufficient mice. J Neurosci. 2013 Mar 20; 33(12):5352-61. PMID: 23516300.
      Citations: 73     Fields:    Translation:AnimalsCells
    57. Li Z, Lu Y, Xu XL, Gao FB. The FTD/ALS-associated RNA-binding protein TDP-43 regulates the robustness of neuronal specification through microRNA-9a in Drosophila. Hum Mol Genet. 2013; 2(22):218-225.
    58. Almeida S, Zhang Z, Coppola G, Mao W, Futai K, Karydas A, Geschwind MD, Tartaglia MC, Gao F, Gianni D, Sena-Esteves M, Geschwind DH, Miller BL, Farese RV, Gao FB. Induced pluripotent stem cell models of progranulin-deficient frontotemporal dementia uncover specific reversible neuronal defects. Cell Rep. 2012 Oct 25; 2(4):789-98. PMID: 23063362.
      Citations: 77     Fields:    Translation:HumansCells
    59. Gonz?lez-P?rez P, Lu Y, Chian RJ, Sapp PC, Tanzi RE, Bertram L, McKenna-Yasek D, Gao FB, Brown RH. Association of UBQLN1 mutation with Brown-Vialetto-Van Laere syndrome but not typical ALS. Neurobiol Dis. 2012 Dec; 48(3):391-8. PMID: 22766032.
      Citations: 10     Fields:    Translation:Humans
    60. Almeida S, Delaloy C, Liu L, Gao FB. Protocols for investigating microRNA functions in human neural progenitor cells. Methods Mol Biol. 2012; (916):387-402.
    61. Gao FB, Taylor JP. RNA-binding proteins in neurological disease. Brain Res. 2012; (1462):1-2.
    62. Gascon E, Gao FB. Cause or Effect: Misregulation of microRNA Pathways in Neurodegeneration. Front Neurosci. 2012; (6):48.
    63. Lee JA, Gao FB. Neuronal Functions of ESCRTs. Exp Neurobiol. 2012; 1(21):9-15.
    64. Sun K, Westholm JO, Tsurudome K, Hagen JW, Lu Y, Kohwi M, Betel D, Gao FB, Haghighi AP, Doe CQ, Lai EC. Neurophysiological defects and neuronal gene deregulation in Drosophila mir-124 mutants. PLoS Genet. 2012; 2(8):e1002515.
    65. Almeida S, Zhou L, Gao FB. Progranulin, a glycoprotein deficient in frontotemporal dementia, is a novel substrate of several protein disulfide isomerase family proteins. PLoS One. 2011; 6(10):e26454. PMID: 22028881.
      Citations: 30     Fields:    Translation:HumansAnimalsCells
    66. Lee JA, Liu L, Javier R, Kreitzer AC, Delaloy C, Gao FB. ESCRT-III subunits Snf7-1 and Snf7-2 differentially regulate transmembrane cargos in hESC-derived human neurons. Mol Brain. 2011 Oct 05; 4:37. PMID: 21975012.
      Citations: 10     Fields:    Translation:HumansAnimalsCells
    67. Kuo TC, Chen CT, Baron D, Onder TT, Loewer S, Almeida S, Weismann CM, Xu P, Houghton JM, Gao FB, Daley GQ, Doxsey S. Midbody accumulation through evasion of autophagy contributes to cellular reprogramming and tumorigenicity. Nat Cell Biol. 2011 Sep 11; 13(10):1214-23. PMID: 21909099.
      Citations: 133     Fields:    Translation:HumansAnimalsCells
    68. Yuva-Aydemir Y, Simkin A, Gascon E, Gao FB. MicroRNA-9: functional evolution of a conserved small regulatory RNA. RNA Biol. 2011 Jul-Aug; 8(4):557-64. PMID: 21697652.
      Citations: 87     Fields:    Translation:HumansAnimals
    69. Xu XL, Zong R, Li Z, Biswas MH, Fang Z, Nelson DL, Gao FB. FXR1P but not FMRP regulates the levels of mammalian brain-specific microRNA-9 and microRNA-124. J Neurosci. 2011; 39(31):13705-13709.
    70. Gao FB. Context-dependent functions of specific microRNAs in neuronal development. Neural Dev. 2010 Oct 01; 5:25. PMID: 20920300.
      Citations: 82     Fields:    Translation:AnimalsCells
    71. Delaloy C, Gao FB. A new role for microRNA-9 in human neural progenitor cells. Cell Cycle. 2010 Aug 01; 9(15):2913-4. PMID: 20676037.
      Citations: 8     Fields:    Translation:HumansAnimalsCells
    72. Jiao J, Herl LD, Farese RV, Gao FB. MicroRNA-29b regulates the expression level of human progranulin, a secreted glycoprotein implicated in frontotemporal dementia. PLoS One. 2010 May 10; 5(5):e10551. PMID: 20479936.
      Citations: 49     Fields:    Translation:HumansAnimalsCells
    73. Delaloy C, Liu L, Lee JA, Su H, Shen F, Yang GY, Young WL, Ivey KN, Gao FB. MicroRNA-9 coordinates proliferation and migration of human embryonic stem cell-derived neural progenitors. Cell Stem Cell. 2010 Apr 02; 6(4):323-35. PMID: 20362537.
      Citations: 172     Fields:    Translation:HumansAnimalsCells
    74. Lee JA, Liu L, Gao FB. Autophagy defects contribute to neurodegeneration induced by dysfunctional ESCRT-III. Autophagy. 2009 Oct; 5(7):1070-2. PMID: 19738424.
      Citations: 19     Fields:    Translation:HumansAnimalsCells
    75. Lu Y, Ferris J, Gao FB. Frontotemporal dementia and amyotrophic lateral sclerosis-associated disease protein TDP-43 promotes dendritic branching. Mol Brain. 2009 Sep 25; 2:30. PMID: 19781077.
      Citations: 75     Fields:    Translation:HumansAnimalsCells
    76. Ahmad ST, Sweeney ST, Lee JA, Sweeney NT, Gao FB. Genetic screen identifies serpin5 as a regulator of the toll pathway and CHMP2B toxicity associated with frontotemporal dementia. Proc Natl Acad Sci U S A. 2009 Jul 21; 106(29):12168-73. PMID: 19581577.
      Citations: 44     Fields:    Translation:AnimalsCells
    77. Lee JA, Gao FB. Inhibition of autophagy induction delays neuronal cell loss caused by dysfunctional ESCRT-III in frontotemporal dementia. J Neurosci. 2009; 26(29):8506-8511.
    78. Lee JA, Gao FB. ESCRT, autophagy, and frontotemporal dementia. BMB Rep. 2008 Dec 31; 41(12):827-32. PMID: 19123971.
      Citations: 14     Fields:    Translation:HumansCells
    79. Xu XL, Li Y, Wang F, Gao FB. The steady-state level of the nervous-system-specific microRNA-124a is regulated by dFMR1 in Drosophila. J Neurosci. 2008 Nov 12; 28(46):11883-9. PMID: 19005053.
      Citations: 64     Fields:    Translation:AnimalsCells
    80. Lu Y, Wang F, Li Y, Ferris J, Lee JA, Gao FB. The Drosophila homologue of the Angelman syndrome ubiquitin ligase regulates the formation of terminal dendritic branches. Hum Mol Genet. 2009 Feb 01; 18(3):454-62. PMID: 18996915.
      Citations: 43     Fields:    Translation:HumansAnimalsCells
    81. Lee JA, Gao FB. Regulation of Abeta pathology by beclin 1: a protective role for autophagy? J Clin Invest. 2008 Jun; 118(6):2015-8. PMID: 18497881.
      Citations: 16     Fields:    Translation:HumansAnimalsCells
    82. Delaloy C, Gao FB. microRNA-9 multitasking near organizing centers. Nat Neurosci. 2008 Jun; 11(6):625-6. PMID: 18506136.
      Citations: 4     Fields:    Translation:AnimalsCells
    83. Lee JA, Gao FB. Roles of ESCRT in autophagy-associated neurodegeneration. Autophagy. 2008 Feb; 4(2):230-2. PMID: 18094607.
      Citations: 27     Fields:    Translation:HumansAnimalsCells
    84. Gao FB. Posttranscriptional control of neuronal development by microRNA networks. Trends Neurosci. 2008 Jan; 31(1):20-6. PMID: 18054394.
      Citations: 62     Fields:    Translation:HumansAnimalsCells
    85. Gao FB. Molecular and cellular mechanisms of dendritic morphogenesis. Curr Opin Neurobiol. 2007 Oct; 17(5):525-32. PMID: 17933513.
      Citations: 30     Fields:    Translation:AnimalsCells
    86. Lee JA, Beigneux A, Ahmad ST, Young SG, Gao FB. ESCRT-III dysfunction causes autophagosome accumulation and neurodegeneration. Curr Biol. 2007; 18(17):1561-1567.
    87. Li Y, Wang F, Lee JA, Gao FB. MicroRNA-9a ensures the precise specification of sensory organ precursors in Drosophila. Genes Dev. 2006 Oct 15; 20(20):2793-805. PMID: 17015424.
      Citations: 156     Fields:    Translation:AnimalsCells
    88. Sweeney NT, Brenman JE, Jan YN, Gao FB. The coiled-coil protein shrub controls neuronal morphogenesis in Drosophila. Curr Biol. 2006; 10(16):1006-1011.
    89. Tassetto M, Gao FB. Transcriptional control of dendritic patterning in Drosophila neurons. Genome Biol. 2006; 7(7):225. PMID: 16879736.
      Citations: 1     Fields:    Translation:AnimalsCells
    90. Li W, Li Y, Gao FB. Abelson, enabled, and p120 catenin exert distinct effects on dendritic morphogenesis in Drosophila. Dev Dyn. 2005 Nov; 234(3):512-22. PMID: 16003769.
      Citations: 25     Fields:    Translation:AnimalsCells
    91. Li W, Wang F, Menut L, Gao FB. BTB/POZ-zinc finger protein abrupt suppresses dendritic branching in a neuronal subtype-specific and dosage-dependent manner. Neuron. 2004 Sep 16; 43(6):823-34. PMID: 15363393.
      Citations: 48     Fields:    Translation:AnimalsCells
    92. Xu K, Bogert BA, Li W, Su K, Lee A, Gao FB. The fragile X-related gene affects the crawling behavior of Drosophila larvae by regulating the mRNA level of the DEG/ENaC protein pickpocket1. Curr Biol. 2004 Jun 22; 14(12):1025-34. PMID: 15202995.
      Citations: 54     Fields:    Translation:AnimalsCells
    93. Lee A, Li W, Xu K, Bogert BA, Su K, Gao FB. Control of dendritic development by the Drosophila fragile X-related gene involves the small GTPase Rac1. Development. 2003 Nov; 130(22):5543-52. PMID: 14530299.
      Citations: 114     Fields:    Translation:AnimalsCells
    94. Li W, Gao FB. Actin filament-stabilizing protein tropomyosin regulates the size of dendritic fields. J Neurosci. 2003 Jul 16; 23(15):6171-5. PMID: 12867499.
      Citations: 15     Fields:    Translation:AnimalsCells
    95. Gao FB, Bogert BA. Genetic control of dendritic morphogenesis in Drosophila. Trends Neurosci. 2003 May; 26(5):262-8. PMID: 12744843.
      Citations: 17     Fields:    Translation:AnimalsCells
    96. Sweeney NT, Li W, Gao FB. Genetic manipulation of single neurons in vivo reveals specific roles of flamingo in neuronal morphogenesis. Dev Biol. 2002 Jul 01; 247(1):76-88. PMID: 12074553.
      Citations: 30     Fields:    Translation:AnimalsCells
    97. Gao FB. Understanding fragile X syndrome: insights from retarded flies. Neuron. 2002 Jun 13; 34(6):859-62. PMID: 12086633.
      Citations: 13     Fields:    Translation:Animals
    98. Tokumoto YM, Apperly JA, Gao FB, Raff MC. Posttranscriptional regulation of p18 and p27 Cdk inhibitor proteins and the timing of oligodendrocyte differentiation. Dev Biol. 2002 May 01; 245(1):224-34. PMID: 11969268.
      Citations: 30     Fields:    Translation:AnimalsCells
    99. Brenman JE, Gao FB, Jan LY, Jan YN. Sequoia, a tramtrack-related zinc finger protein, functions as a pan-neural regulator for dendrite and axon morphogenesis in Drosophila. Dev Cell. 2001; 5(1):667-677.
    100. Gao FB, Kohwi M, Brenman JE, Jan LY, Jan YN. Control of dendritic field formation in Drosophila: the roles of flamingo and competition between homologous neurons. Neuron. 2000 Oct; 28(1):91-101. PMID: 11086986.
      Citations: 59     Fields:    Translation:AnimalsCells
    101. Gao FB, Brenman JE, Jan LY, Jan YN. Genes regulating dendritic outgrowth, branching, and routing in Drosophila. Genes Dev. 1999 Oct 01; 13(19):2549-61. PMID: 10521399.
      Citations: 129     Fields:    Translation:AnimalsCells
    102. Raff MC, Durand B, Gao FB. Cell number control and timing in animal development: the oligodendrocyte cell lineage. Int J Dev Biol. 1998; 42(3):263-7. PMID: 9654007.
      Citations: 22     Fields:    Translation:AnimalsCells
    103. Gao FB, Apperly J, Raff M. Cell-intrinsic timers and thyroid hormone regulate the probability of cell-cycle withdrawal and differentiation of oligodendrocyte precursor cells. Dev Biol. 1998; 1(197):54-66.
    104. Gao FB. Messenger RNAs in dendrites: localization, stability, and implications for neuronal function. Bioessays. 1998 Jan; 20(1):70-8. PMID: 9504049.
      Citations: 10     Fields:    Translation:HumansAnimalsCells
    105. Durand B, Gao FB, Raff M. Accumulation of the cyclin-dependent kinase inhibitor p27/Kip1 and the timing of oligodendrocyte differentiation. EMBO J. 1997 Jan 15; 16(2):306-17. PMID: 9029151.
      Citations: 83     Fields:    Translation:AnimalsCells
    106. Gao FB, Durand B, Raff M. Oligodendrocyte precursor cells count time but not cell divisions before differentiation. Curr Biol. 1997; 2(7):152-155.
    107. Gao FB, Raff M. Cell size control and a cell-intrinsic maturation program in proliferating oligodendrocyte precursor cells. J Cell Biol. 1997; 6(138):1367-1377.
    108. Gao FB, Keene JD. Hel-N1/Hel-N2 proteins are bound to poly(A)+ mRNA in granular RNP structures and are implicated in neuronal differentiation. J Cell Sci. 1996 Mar; 109 ( Pt 3):579-89. PMID: 8907704.
      Citations: 46     Fields:    Translation:HumansAnimalsCells
    109. Gao FB, Carson CC, Levine T, Keene JD. Selection of a subset of mRNAs from combinatorial 3' untranslated region libraries using neuronal RNA-binding protein Hel-N1. Proc Natl Acad Sci U S A. 1994; 23(91):1207-1211.
    110. Levine TD, Gao F, King PH, Andrews LG, Keene JD. Hel-N1: an autoimmune RNA-binding protein with specificity for 3' uridylate-rich untranslated regions of growth factor mRNAs. Mol Cell Biol. 1993; 6(13):3494-3504.
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