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Fen-Biao Gao PhD

Endowed TitleGovernor Paul Cellucci Chair in Neuroscience Research
InstitutionUMass Chan Medical School
DepartmentRNA Therapeutics Institute
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
    2017 - NowGovernor Paul Cellucci Chair in Neuroscience Research, UMMS
    2018 - 2024Javits 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.
    Newest   |   Oldest   |   Most Cited   |   Most Discussed   |   Timeline   |   Field Summary   |   Plain Text
    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. Lorenzini I, Alsop E, Levy J, Gittings LM, Lall D, Rabichow BE, Moore S, Pevey R, Bustos LM, Burciu C, Bhatia D, Singer M, Saul J, McQuade A, Tzioras M, Mota TA, Logemann A, Rose J, Almeida S, Gao FB, Marks M, Donnelly CJ, Hutchins E, Hung ST, Ichida J, Bowser R, Spires-Jones T, Blurton-Jones M, Gendron TF, Baloh RH, Van Keuren-Jensen K, Sattler R. Moderate intrinsic phenotypic alterations in C9orf72 ALS/FTD iPSC-microglia despite the presence of C9orf72 pathological features. Front Cell Neurosci. 2023; 17:1179796. PMID: 37346371.
    2. Hung ST, Linares GR, Chang WH, Eoh Y, Krishnan G, Mendonca S, Hong S, Shi Y, Santana M, Kueth C, Macklin-Isquierdo S, Perry S, Duhaime S, Maios C, Chang J, Perez J, Couto A, Lai J, Li Y, Alworth SV, Hendricks E, Wang Y, Zlokovic BV, Dickman DK, Parker JA, Zarnescu DC, Gao FB, Ichida JK. PIKFYVE inhibition mitigates disease in models of diverse forms of ALS. Cell. 2023 02 16; 186(4):786-802.e28. PMID: 36754049.
      Citations: 13     Fields:    Translation:AnimalsCells
    3. Phillips HL, Dai H, Choi SY, Jansen-West K, Zajicek AS, Daly L, Petrucelli L, Gao FB, Yao WD. Dorsomedial prefrontal hypoexcitability underlies lost empathy in frontotemporal dementia. Neuron. 2023 03 15; 111(6):797-806.e6. PMID: 36638803.
      Citations: 2     Fields:    Translation:AnimalsCells
    4. Arredondo C, Cefaliello C, Dyrda A, Jury N, Martinez P, D?az I, Amaro A, Tran H, Morales D, Pertusa M, Stoica L, Fritz E, Corval?n D, Abarz?a S, M?ndez-Ruette M, Fern?ndez P, Rojas F, Kumar MS, Aguilar R, Almeida S, Weiss A, Bustos FJ, Gonz?lez-Nilo F, Otero C, Tevy MF, Bosco DA, S?ez JC, K?hne T, Gao FB, Berry JD, Nicholson K, Sena-Esteves M, Madrid R, Varela D, Montecino M, Brown RH, van Zundert B. Excessive release of inorganic polyphosphate by ALS/FTD astrocytes causes non-cell-autonomous toxicity to motoneurons. Neuron. 2022 05 18; 110(10):1656-1670.e12. PMID: 35276083.
      Citations: 16     Fields:    Translation:HumansAnimalsCells
    5. Sonobe Y, Aburas J, Krishnan G, Fleming AC, Ghadge G, Islam P, Warren EC, Gu Y, Kankel MW, Brown AEX, Kiskinis E, Gendron TF, Gao FB, Roos RP, Kratsios P. A C. elegans model of C9orf72-associated ALS/FTD uncovers a conserved role for eIF2D in RAN translation. Nat Commun. 2021 10 15; 12(1):6025. PMID: 34654821.
      Citations: 17     Fields:    Translation:HumansAnimalsCells
    6. 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: 59     Fields:    Translation:AnimalsCells
    7. 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: 18     Fields:    Translation:HumansAnimalsCells
    8. 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: 68     Fields:    Translation:HumansAnimalsCells
    9. 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
    10. 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
    11. 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: 351     Fields:    Translation:HumansAnimalsCells
    12. Gao FB, Richter JD, Cleveland DW. Rethinking Unconventional Translation in Neurodegeneration. Cell. 2017 Nov 16; 171(5):994-1000. PMID: 29149615.
      Citations: 39     Fields:    Translation:HumansAnimalsCells
    13. 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: 97     Fields:    Translation:HumansAnimals
    14. 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: 49     Fields:    Translation:HumansCells
    15. 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: 58     Fields:    Translation:HumansCells
    16. 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
    17. Lee JA, Yue Z, Gao FB. Autophagy in neurodegenerative diseases. Brain Res. 2016 10 15; 1649(Pt B):141-142. PMID: 27712723.
      Citations: 5     Fields:    Translation:HumansAnimals
    18. 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: 217     Fields:    Translation:HumansCells
    19. 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: 45     Fields:    Translation:HumansCells
    20. 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
    21. 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: 78     Fields:    Translation:HumansAnimalsCells
    22. 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: 22     Fields:    
    23. 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: 7     Fields:    Translation:Humans
    24. 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: 8     Fields:    Translation:HumansCells
    25. 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: 43     Fields:    Translation:HumansAnimalsCells
    26. 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: 60     Fields:    Translation:HumansAnimalsCells
    27. 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.
    28. 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).
    29. 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: 81     Fields:    Translation:HumansAnimalsCells
    30. 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: 24     Fields:    Translation:AnimalsCells
    31. 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: 45     Fields:    Translation:AnimalsCells
    32. 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: 92     Fields:    Translation:AnimalsCells
    33. Gao FB, Taylor JP. RNA metabolism in neurological disease. Brain Res. 2014 Oct 10; 1584:1-2. PMID: 25248315.
      Citations: 2     Fields:    Translation:Humans
    34. 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
    35. 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: 45     Fields:    Translation:HumansCells
    36. 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
    37. 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: 72     Fields:    Translation:HumansCells
    38. 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: 35     Fields:    Translation:HumansAnimalsCells
    39. 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: 188     Fields:    Translation:HumansAnimalsCells
    40. 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: 79     Fields:    Translation:AnimalsCells
    41. 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.
    42. 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: 81     Fields:    Translation:HumansCells
    43. 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
    44. 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.
    45. Lee JA, Gao FB. Neuronal Functions of ESCRTs. Exp Neurobiol. 2012; 1(21):9-15.
    46. Gascon E, Gao FB. Cause or Effect: Misregulation of microRNA Pathways in Neurodegeneration. Front Neurosci. 2012; (6):48.
    47. Gao FB, Taylor JP. RNA-binding proteins in neurological disease. Brain Res. 2012; (1462):1-2.
    48. 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.
    49. 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: 31     Fields:    Translation:HumansAnimalsCells
    50. 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: 11     Fields:    Translation:HumansAnimalsCells
    51. 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: 93     Fields:    Translation:HumansAnimals
    52. 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.
    53. Gao FB. Context-dependent functions of specific microRNAs in neuronal development. Neural Dev. 2010 Oct 01; 5:25. PMID: 20920300.
      Citations: 84     Fields:    Translation:AnimalsCells
    54. 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
    55. 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: 52     Fields:    Translation:HumansAnimalsCells
    56. 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):323-335. PMID: 20362537.
      Citations: 178     Fields:    Translation:HumansAnimalsCells
    57. 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: 20     Fields:    Translation:HumansAnimalsCells
    58. 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: 76     Fields:    Translation:HumansAnimalsCells
    59. 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: 46     Fields:    Translation:AnimalsCells
    60. 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.
    61. 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
    62. 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: 65     Fields:    Translation:AnimalsCells
    63. 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: 44     Fields:    Translation:HumansAnimalsCells
    64. 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
    65. 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
    66. 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
    67. Gao FB. Posttranscriptional control of neuronal development by microRNA networks. Trends Neurosci. 2008 Jan; 31(1):20-6. PMID: 18054394.
      Citations: 64     Fields:    Translation:HumansAnimalsCells
    68. 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
    69. 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.
    70. 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: 157     Fields:    Translation:AnimalsCells
    71. 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.
    72. 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
    73. 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
    74. 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: 49     Fields:    Translation:AnimalsCells
    75. 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
    76. 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
    77. 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
    78. 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
    79. 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
    80. 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
    81. 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
    82. 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.
    83. 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
    84. 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: 132     Fields:    Translation:AnimalsCells
    85. 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
    86. 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
    87. 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.
    88. 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
    89. Gao FB, Durand B, Raff M. Oligodendrocyte precursor cells count time but not cell divisions before differentiation. Curr Biol. 1997; 2(7):152-155.
    90. 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.
    91. 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
    92. 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.
    93. 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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