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  1. Sattler R, Traynor BJ, Robertson J, Van Den Bosch L, Barmada SJ, Svendsen CN, Disney MD, Gendron TF, Wong PC, Turner MR, Boxer A, Babu S, Benatar M, Kurnellas M, Rohrer JD, Donnelly CJ, Bustos LM, Van Keuren-Jensen K, Dacks PA, Sabbagh MN; Attendees of the inaugural C9ORF72 FTD/ALS Summit. Roadmap for C9ORF72 in Frontotemporal Dementia and Amyotrophic Lateral Sclerosis: Report on the C9ORF72 FTD/ALS Summit. Neurol Ther. 2023 Oct 17. doi: 10.1007/s40120-023-00548-8. Online ahead of print. Pubmed
  2. Fang M, Deibler SK, Nana AL, Vatsavayai SC, Banday S, Zhou Y, Almeida S, Weiss A, Brown RH, Seeley W, Gao FB, Green MR. Loss of TDP-43 function contributes to genomic instability in Amyotrophic Lateral Sclerosis. Frontiers in Neuroscience, sec Neurodegeneration. 2023 Oct 2; Volume 17 - 2023 | Pubmed

  3. Sonobe Y, Lee S, Krishnan G, Gu Y, Kwon DY, Gao FB, Roos RP*, Kratsios P*. Translation of dipeptide repeat proteins in C9ORF72 ALS/FTD through unique and redundant AUG initiation codons. Elife. 2023 Sep 7;12:e83189. doi: 10.7554/eLife.83189. (Online ahead of print). (*:co-corresponding authors) Pubmed

  4. Rojas F, Aguilar R, Almeida S, Fritz E, Corvalán D, Ampuero E, Abarzúa S, Garcés P, Amaro A, Diaz I, Arredondo C, Cortes N, Sanchez M, Mercado C, Varela-Nallar L, Gao FB, Montecino M, van Zundert B. Mature iPSC-derived astrocytes of an ALS/FTD patient carrying the TDP43A90Vmutation display a mild reactive state and release polyP toxic to motoneurons.Front Cell Dev Biol. 2023 Jul 28;11:1226604. Pubmed

  5. 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 Jun 6;17:1179796. Pubmed

  6. Halim DO, Munson M, Gao FB. The exocyst complex in neurological disorders. Hum Genet. 2023 Apr 22. doi: 10.1007/s00439-023-02558-w. (Online ahead of print). Review Pubmed
  7. 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. (Online ahead of print). Pubmed
  8. Jun YB, 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. doi: 10.1080/15548627.2023.2169309. (Online ahead of print). Pubmed
  9. 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 Jan 9;S0896-6273(22)01124-2. (Online ahead of print). Pubmed
  10. 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 Feb 2:S0092-8674(23)00005-3. doi: 10.1016/j.cell.2023.01.005. (Online ahead of print). Pubmed
  1. 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 Nov 22;10(1):169. Pubmed
  2. 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. Nature Commun. 2022 Oct 21, 13:6286. (*:co-corresponding authors). Pubmed
  3. 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. Nature Commun2022 May 19, 13:2799. (*:co-corresponding authors).Pubmed
  4. Loveland AB, Svidritskiy E, Susorov D, Lee S, Park A, Zvornicanin S, Demo G, Gao FB*, Korostelev AA*. Ribosome inhibition by C9ORF72-ALS/FTDassociated poly-PR and poly-GR proteins revealed by cryo-EM. Nature Commun. 2022 May 19, 13:2776 (*:co-corresponding authors).  Pubmed
  5. Arredondo C, Cefaliello C, Dyrda A, Jury N, Martinez P, Díaz I, Amaro A, Tran H, Morales D, Pertusa M, Stoica L, Fritz E, Corvalán D, Abarzúa S, Méndez-Ruette M, Fernández P, Rojas F, Kumar MS, Aguilar R, Almeida S, Weiss A, Bustos FJ, González-Nilo F, Otero C, Tevy MF, Bosco DA, Sáez JC, Kähne 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 phosphate by ALS/FTD astrocytes causes non-cell-autonomous toxicity to motoneurons. Neuron. 2022 Mar 3, S0896-6273(22)00148-9. Pubmed
  6. Halim D, Gao FB. RNA targets of TDP-43: Which one is more important in neurodegeneration? Transl Neurodegener. 2022 Feb 25;11(1):12. Pubmed
  1. 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. 2021 Nov 30, rna.078963.121. (#: co-first authors; *:co-corresponding authors). Pubmed
  2. 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 Oct 15, 12(1):6025. (*:co-corresponding authors). Pubmed
  3. Lampasona A, Almeida S, Gao F-B. Translation of the poly(GR) frame in C9ORF72-ALS/FTD is regulated by cis-elements involved in alternative splicing.  Neurobiol Aging. 2021 105:327–332. Pubmed
  4. Lu Y, Almeida S, Gao FB. TBK1 haploinsufficiency in ALS and FTD compromises membrane trafficking. Acta Neuropathol. 2021 Jul; 142(1): 217–221. PMID: 34081168. Pubmed
  5. 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 Feb 4;184(3):689-708.e20. PMID: 33482083. Pubmed
  1. 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 Aug 26;10(1):14221. PMID: 32848189. Pubmed
  2. 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 Aug 4;32(5):107989. PMID: 32755582. Pubmed
  3. 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 Apr 7. (#: co-first authors;  *: co-corresponding authors). PMID:32266467. Pubmed 
  4. Frew J, Baradaran-Heravi A, Balgi AD, Wu X, Yan TD, Arns S, Shidmoossavee FS, Tan J, Jaquith JB, Jansen-West KR, Lynn FC, Gao FB, Petrucelli L, Feldman HH, Mackenzie IR, Roberge M, Nygaard HB. Premature termination codon readthrough upregulates progranulin expression and improves lysosomal function in preclinical models of GRN deficiency. Mol Neurodegener. 2020 Mar 16;15(1):21. PMID: 32178712. Pubmed
  1. 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 Nov 29;10(1):5466. (#: co-first author;  *: co-corresponding author). PMID: 31784536. View in: Pubmed 
  2. 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 Oct 17. [Epub ahead of print]. (*: co-corresponding author). PMID: 31624870. View in: Pubmed 
  3. 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 Sep 19. [Epub ahead of print] PMID:31587919. View in: Pubmed
  4. 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 May 13. [Epub ahead of print] PMID:31086314. View in: Pubmed
  5. 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 USA. 2019 Apr 24. pii: 201901313. [Epub ahead of print]. View in: Pubmed
  1. Yuva-Aydemir Y, Almeida S, Gao FB. Insights into C9ORF72-Related ALS/FTD from Drosophila and iPSC Models. Trends Neurosci. 2018 May 2. pii: S0166-2236(18)30087-0. Review. PMID: 29729808. View in: Pubmed
  2. Liu X, He J, Gao FB, Gitler AD, Fan D. The epidemiology and genetics of Amyotrophic lateral sclerosis in China. Brain Res. 2018 Mar 1. pii: S0006-8993(18)30105-7. PMID: 29501653. View in: Pubmed
  3. 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 Feb 8. PMID: 29432529. View in: Pubmed
  4. 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, Lécuyer E, Yeo GW. Context-Dependent and Disease-Specific Diversity in Protein Interactions within Stress Granules. Cell. 2018 Jan 25;172(3):590-604.e13. View in: Pubmed
  1. Gao FB, Richter JD, Cleveland DW. Rethinking Unconventional Translation in Neurodegeneration. Cell. 2017 Nov 16; 171(5):994-1000. PMID: 29149615. View in: Pubmed
  2. Gao FB, Almeida S, Lopez-Gonzalez R. Dysregulated molecular pathways in amyotrophic lateral sclerosis-frontotemporal dementia spectrum disorder. EMBO J. 2017 Oct 16; 36(20):2931-2950. PMID: 28916614. View in: PubMed
  3. 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 proteaseMol Neurodegener. 2017 Jul 25;12(1):55. View in: PubMed
  4. 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 Jun 13; 19(11):2244-2256. PMID: 28614712. View in: PubMed
  5. 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 Apr 03. PMID: 28379367. View in: PubMed
  6. Gao FB, Richter JD. Microsatellite Expansion Diseases: Repeat Toxicity Found in Translation. Neuron. 2017 Jan 18;93(2):249-251. View in: PubMed
  1. Lopez-Gonzalez RLu YGendron TFKarydas ATran HYang DPetrucelli LMiller BLAlmeida SGao 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. View in: PubMed
  2. Lee JA, Yue Z, Gao FB. Autophagy in neurodegenerative diseases. Brain Res. 2016 Oct 15; 1649(Pt B):141-142. View in: PubMed
  3. Silva MC, Cheng C, Mair W, Almeida S, Fong H, Biswas MH, 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 Sep 13; 7(3):325-40. View in: PubMed
  4. Biswas MH, 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 Sep 13; 7(3):316-24. View in: PubMed
  5. 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. View in: PubMed
  6. 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 Jul 1. pii: sctm.2015-0340.  View in: PubMed
  7. Almeida S, Gao FB. Lost & found: C9ORF72 and the autophagy pathway in ALS/FTD. EMBO J. 2016 Jun 15; 35(12):1251-3. View in: PubMed
  8. 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 Jun; 42:35-40. View in: PubMed
  9. Woehlbier U, Colombo A, Saaranen MJ, Pérez 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, Muñoz 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. View in: PubMed
  1. Schweizer Burguete A, 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; 4. View in: PubMed
  2. 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 2; 88(5):902-9. View in: PubMed
  3. 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. View in: PubMed
  4. 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). View in: PubMed
  5. 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. View in: PubMed
  6. 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. View in: PubMed
  7. 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. View in: PubMed


  1. 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. View in: PubMed
  2. Gao FB, Taylor JP. RNA metabolism in neurological disease. Brain Res. 2014 Oct 10; 1584:1-2. View in: PubMed
  3. 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. View in: PubMed
  4. 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. View in: PubMed
  5. 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. View in: PubMed
  6. 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. View in: PubMed


  1. 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. View in: PubMed
  2. 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. View in: PubMed
  3. 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. View in: PubMed
  4. 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. View in: PubMed
  5. 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. View in: PubMed


  1. 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. View in: PubMed
  2. González-Pérez 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. View in: PubMed
  3. 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. View in: PubMed
  4. Lee JA, Gao FB. Neuronal Functions of ESCRTs. Exp Neurobiol. 2012; 1(21):9-15. View in: PubMed
  5. Gascon E, Gao FB. Cause or Effect: Misregulation of microRNA Pathways in Neurodegeneration. Front Neurosci. 2012; (6):48. View in: PubMed
  6. Gao FB, Taylor JP. RNA-binding proteins in neurological disease. Brain Res. 2012; (1462):1-2. View in: PubMed
  7. 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. View in: PubMed


  1. 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. View in: PubMed
  2. 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; 4:37. View in: PubMed
  3. 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 Oct; 13(10):1214-23. View in: PubMed
  4. 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. View in: PubMed
  5. 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. View in: PubMed


  1. Gao FB. Context-dependent functions of specific microRNAs in neuronal development. Neural Dev. 2010; 5:25. View in: PubMed
  2. Delaloy C, Gao FB. A new role for microRNA-9 in human neural progenitor cells. Cell Cycle. 2010 Aug 1; 9(15):2913-4. View in: PubMed
  3. 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; 5(5):e10551. View in: PubMed
  4. 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 2; 6(4):323-35. View in: PubMed


  1. Lee JA, Liu L, Gao FB. Autophagy defects contribute to neurodegeneration induced by dysfunctional ESCRT-III. Autophagy. 2009 Oct; 5(7):1070-2. View in: PubMed
  2. Lu Y, Ferris J, Gao FB. Frontotemporal dementia and amyotrophic lateral sclerosis-associated disease protein TDP-43 promotes dendritic branching. Mol Brain. 2009; 2:30. View in: PubMed
  3. 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. View in: PubMed
  4. 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. View in: PubMed
  5. 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 1; 18(3):454-62.View in: PubMed


  1. Lee JA, Gao FB. ESCRT, autophagy, and frontotemporal dementia. BMB Rep. 2008 Dec 31; 41(12):827-32. View in: PubMed
  2. 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. View in: PubMed
  3. 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. View in: PubMed
  4. Delaloy C, Gao FB. microRNA-9 multitasking near organizing centers. Nat Neurosci. 2008 Jun; 11(6):625-6. View in: PubMed
  5. Lee JA, Gao FB. Roles of ESCRT in autophagy-associated neurodegeneration. Autophagy. 2008 Feb; 4(2):230-2. View in: PubMed
  6. Gao FB. Posttranscriptional control of neuronal development by microRNA networks. Trends Neurosci. 2008 Jan; 31(1):20-6. View in: PubMed


  1. Gao FB. Molecular and cellular mechanisms of dendritic morphogenesis. Curr Opin Neurobiol. 2007 Oct; 17(5):525-32. View in: PubMed
  2. 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. View in: PubMed


  1. 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. View in: PubMed
  2. 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.  View in: PubMed
  3. Tassetto M, Gao FB. Transcriptional control of dendritic patterning in Drosophila neurons. Genome Biol. 2006; 7(7):225. View in: PubMed


  1. 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. View in: PubMed


  1. 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. View in: PubMed
  2. 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. View in: PubMed


  1. 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. View in: PubMed
  2. Li W, Gao FB. Actin filament-stabilizing protein tropomyosin regulates the size of dendritic fields. J Neurosci. 2003 Jul 16; 23(15):6171-5. View in: PubMed
  3. Gao FB, Bogert BA. Genetic control of dendritic morphogenesis in Drosophila. Trends Neurosci. 2003 May; 26(5):262-8. View in: PubMed


  1. 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 1; 247(1):76-88. View in: PubMed
  2. Gao FB. Understanding fragile X syndrome: insights from retarded flies. Neuron. 2002 Jun 13; 34(6):859-62. View in: PubMed
  3. 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 1; 245(1):224-34. View in: PubMed


2001 and before
  1. 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. View in: PubMed
  2. 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. View in: PubMed
  3. Gao FB, Brenman JE, Jan LY, Jan YN. Genes regulating dendritic outgrowth, branching, and routing in Drosophila. Genes Dev. 1999 Oct 1; 13(19):2549-61. View in: PubMed
  4. Gao FB. Messenger RNAs in dendrites: localization, stability, and implications for neuronal function. Bioessays. 1998 Jan; 20(1):70-8. View in: PubMed
  5. 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. View in: PubMed
  6. 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. View in: PubMed
  7. 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. View in: PubMed
  8. Gao FB, Durand B, Raff M. Oligodendrocyte precursor cells count time but not cell divisions before differentiation. Curr Biol. 1997; 2(7):152-155. View in: PubMed
  9. 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.  View in: PubMed
  10. 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. View in: PubMed
  11. 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. View in: PubMed
  12. 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. View in: PubMed