Stephanie Cherqui

Title(s)Associate Professor In Residence, Pediatrics
SchoolHealth Sciences
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    Collapse Research 
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    Hematopoietic Stem Cell Gene Therapy for Friedreich's ataxia
    NIH/NINDS R01NS108965Sep 1, 2018 - Jun 30, 2023
    Role: Principal Investigator
    Chaperone-mediated autophagy in corneal cystinosis
    NIH/NEI R21EY028642Feb 1, 2018 - Jan 31, 2020
    Role: Co-Principal Investigator
    Molecular and Cellular Mechanisms of the Lysosomal Storage Disease Cystinosis
    NIH/NIDDK R01DK110162Aug 9, 2017 - Apr 30, 2020
    Role: Co-Principal Investigator
    Hematopoietic stem cell-based therapy for Friedrich Ataxia
    NIH/NINDS R21NS090066Aug 15, 2014 - Jul 31, 2016
    Role: Principal Investigator
    Toxicology studies for gene-modified stem cell transplantation for cystinosis
    NIH/NIDDK R01DK099338Aug 6, 2013 - May 31, 2015
    Role: Principal Investigator
    Kidney-targeted gene delivery for cystinosis
    NIH/NIDDK R21DK090548Sep 1, 2011 - Aug 31, 2013
    Role: Principal Investigator
    Lentiviral-transduced hematopoictic stem cell transplantation for cystinosis
    NIH/NIDDK R01DK090058Jan 1, 2011 - Jan 31, 2020
    Role: Principal Investigator

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    Collapse Publications
    Publications listed below are automatically derived from MEDLINE/PubMed and other sources, which might result in incorrect or missing publications. Researchers can login to make corrections and additions, or contact us for help.
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    1. Rocca CJ, Cherqui S. Potential use of stem cells as a therapy for cystinosis. Pediatr Nephrol. 2018 May 22. PMID: 29789935.
      View in: PubMed
    2. Rocca CJ, Goodman SM, Dulin JN, Haquang JH, Gertsman I, Blondelle J, Smith JLM, Heyser CJ, Cherqui S. Transplantation of wild-type mouse hematopoietic stem and progenitor cells ameliorates deficits in a mouse model of Friedreich's ataxia. Sci Transl Med. 2017 Oct 25; 9(413). PMID: 29070698.
      View in: PubMed
    3. Ariazi J, Benowitz A, De Biasi V, Den Boer ML, Cherqui S, Cui H, Douillet N, Eugenin EA, Favre D, Goodman S, Gousset K, Hanein D, Israel DI, Kimura S, Kirkpatrick RB, Kuhn N, Jeong C, Lou E, Mailliard R, Maio S, Okafo G, Osswald M, Pasquier J, Polak R, Pradel G, de Rooij B, Schaeffer P, Skeberdis VA, Smith IF, Tanveer A, Volkmann N, Wu Z, Zurzolo C. Tunneling Nanotubes and Gap Junctions-Their Role in Long-Range Intercellular Communication during Development, Health, and Disease Conditions. Front Mol Neurosci. 2017; 10:333. PMID: 29089870.
      View in: PubMed
    4. Zhang J, Johnson JL, He J, Napolitano G, Ramadass M, Rocca C, Kiosses WB, Bucci C, Xin Q, Gavathiotis E, Cuervo AM, Cherqui S, Catz SD. Cystinosin, the small GTPase Rab11, and the Rab7 effector RILP regulate intracellular trafficking of the chaperone-mediated autophagy receptor LAMP2A. J Biol Chem. 2017 06 23; 292(25):10328-10346. PMID: 28465352.
      View in: PubMed
    5. Cherqui S, Courtoy PJ. The renal Fanconi syndrome in cystinosis: pathogenic insights and therapeutic perspectives. Nat Rev Nephrol. 2017 Feb; 13(2):115-131. PMID: 27990015.
      View in: PubMed
    6. Rocca CJ, Kreymerman A, Ur SN, Frizzi KE, Naphade S, Lau A, Tran T, Calcutt NA, Goldberg JL, Cherqui S. Treatment of Inherited Eye Defects by Systemic Hematopoietic Stem Cell Transplantation. Invest Ophthalmol Vis Sci. 2015 Nov; 56(12):7214-23. PMID: 26540660; PMCID: PMC4640476.
    7. Cheung WW, Cherqui S, Ding W, Esparza M, Zhou P, Shao J, Lieber RL, Mak RH. Muscle wasting and adipose tissue browning in infantile nephropathic cystinosis. J Cachexia Sarcopenia Muscle. 2016 05; 7(2):152-64. PMID: 27493869.
      View in: PubMed
    8. Napolitano G, Johnson JL, He J, Rocca CJ, Monfregola J, Pestonjamasp K, Cherqui S, Catz SD. Impairment of chaperone-mediated autophagy leads to selective lysosomal degradation defects in the lysosomal storage disease cystinosis. EMBO Mol Med. 2015 Feb; 7(2):158-74. PMID: 25586965; PMCID: PMC4328646.
    9. Naphade S, Sharma J, Gaide Chevronnay HP, Shook MA, Yeagy BA, Rocca CJ, Ur SN, Lau AJ, Courtoy PJ, Cherqui S. Brief reports: Lysosomal cross-correction by hematopoietic stem cell-derived macrophages via tunneling nanotubes. Stem Cells. 2015 Jan; 33(1):301-9. PMID: 25186209; PMCID: PMC4270893.
    10. Emma F, Nesterova G, Langman C, Labbé A, Cherqui S, Goodyer P, Janssen MC, Greco M, Topaloglu R, Elenberg E, Dohil R, Trauner D, Antignac C, Cochat P, Kaskel F, Servais A, Wühl E, Niaudet P, Van't Hoff W, Gahl W, Levtchenko E. Nephropathic cystinosis: an international consensus document. Nephrol Dial Transplant. 2014 Sep; 29 Suppl 4:iv87-94. PMID: 25165189; PMCID: PMC4158338.
    11. Prencipe G, Caiello I, Cherqui S, Whisenant T, Petrini S, Emma F, De Benedetti F. Inflammasome activation by cystine crystals: implications for the pathogenesis of cystinosis. J Am Soc Nephrol. 2014 Jun; 25(6):1163-9. PMID: 24525029; PMCID: PMC4033370.
    12. Cherqui S. Is genetic rescue of cystinosis an achievable treatment goal? Nephrol Dial Transplant. 2014 Mar; 29(3):522-8. PMID: 23861466; PMCID: PMC3938296.
    13. Johnson JL, Napolitano G, Monfregola J, Rocca CJ, Cherqui S, Catz SD. Upregulation of the Rab27a-dependent trafficking and secretory mechanisms improves lysosomal transport, alleviates endoplasmic reticulum stress, and reduces lysosome overload in cystinosis. Mol Cell Biol. 2013 Aug; 33(15):2950-62. PMID: 23716592; PMCID: PMC3719680.
    14. Harrison F, Yeagy BA, Rocca CJ, Kohn DB, Salomon DR, Cherqui S. Hematopoietic stem cell gene therapy for the multisystemic lysosomal storage disorder cystinosis. Mol Ther. 2013 Feb; 21(2):433-44. PMID: 23089735; PMCID: PMC3594011.
    15. Cherqui S. Cysteamine therapy: a treatment for cystinosis, not a cure. Kidney Int. 2012 Jan; 81(2):127-9. PMID: 22205430; PMCID: PMC3386783.
    16. Taniguchi N, Caramés B, Hsu E, Cherqui S, Kawakami Y, Lotz M. Expression patterns and function of chromatin protein HMGB2 during mesenchymal stem cell differentiation. J Biol Chem. 2011 Dec 02; 286(48):41489-98. PMID: 21890638; PMCID: PMC3308860.
    17. Simpson J, Nien CJ, Flynn K, Jester B, Cherqui S, Jester J. Quantitative in vivo and ex vivo confocal microscopy analysis of corneal cystine crystals in the Ctns knockout mouse. Mol Vis. 2011; 17:2212-20. PMID: 21897743; PMCID: PMC3164685.
    18. Yeagy BA, Cherqui S. Kidney repair and stem cells: a complex and controversial process. Pediatr Nephrol. 2011 Sep; 26(9):1427-34. PMID: 21336814.
      View in: PubMed
    19. Yeagy BA, Harrison F, Gubler MC, Koziol JA, Salomon DR, Cherqui S. Kidney preservation by bone marrow cell transplantation in hereditary nephropathy. Kidney Int. 2011 Jun; 79(11):1198-206. PMID: 21248718.
      View in: PubMed
    20. Syres K, Harrison F, Tadlock M, Jester JV, Simpson J, Roy S, Salomon DR, Cherqui S. Successful treatment of the murine model of cystinosis using bone marrow cell transplantation. Blood. 2009 Sep 17; 114(12):2542-52. PMID: 19506297.
      View in: PubMed
    21. Cherqui S, Kingdon KM, Thorpe C, Kurian SM, Salomon DR. Lentiviral gene delivery of vMIP-II to transplanted endothelial cells and endothelial progenitors is proangiogenic in vivo. Mol Ther. 2007 Jul; 15(7):1264-72. PMID: 17505479.
      View in: PubMed
    22. Cherqui S, Kurian SM, Schussler O, Hewel JA, Yates JR, Salomon DR. Isolation and angiogenesis by endothelial progenitors in the fetal liver. Stem Cells. 2006 Jan; 24(1):44-54. PMID: 16099996.
      View in: PubMed
    23. Martina Y, Kurian S, Cherqui S, Evanoff G, Wilson C, Salomon DR. Pseudotyping of porcine endogenous retrovirus by xenotropic murine leukemia virus in a pig islet xenotransplantation model. Am J Transplant. 2005 Aug; 5(8):1837-47. PMID: 15996230.
      View in: PubMed