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Melissa J Spencer

Title(s)Professor, Neurology
SchoolMedicine
Address760 Westwood Plaza 477
Los Angeles CA 90024
Phone3107945225
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    Optimizing and validation of gene therapy vectors to treat limb girdle muscular dystophy
    NIH/NINDS R01NS117912Aug 1, 2020 - Apr 30, 2025
    Role: Principal Investigator
    FASEB SRC on Calpains in Health and Disease
    NIH/NINDS R13NS111915Jul 1, 2019 - Jun 30, 2020
    Role: Principal Investigator
    Identification of Enhancers of Therapeutic Exon Skipping for DMD
    NIH/NIAMS RC1AR058333Sep 25, 2009 - Aug 31, 2011
    Role: Co-Principal Investigator
    UCLA Muscular Dystrophy Core Center
    NIH/NIAMS P30AR057230Apr 1, 2009 - Mar 31, 2019
    Role: Principal Investigator
    Pathogenic Mechanisms in Limb Girdle Muscular Dystrophies
    NIH/NIAMS R01AR052693Aug 10, 2007 - Jul 31, 2013
    Role: Principal Investigator
    FASEB Summer Conference on: The Biology of Calpains in Health and Disease
    NIH/NIAMS R13AR055422Jul 1, 2007 - Jun 30, 2008
    Role: Principal Investigator
    Mechanisms Underlying Limb Girdle Muscular Dystrophy2A Due to Calpain 3 Mutations
    NIH/NIAMS R01AR048177Sep 24, 2001 - Aug 31, 2017
    Role: Principal Investigator
    THERAPEUTIC APPROACHES FOR MUSCULAR DYSTROPHY
    NIH/NIAMS R01AR046911May 5, 2000 - Apr 30, 2008
    Role: Principal Investigator
    CALPAIN REGULATION OF MUSCLE NECROSIS AND REGENERATION
    NIH/NIAMS R03AR045838Sep 30, 1998 - Aug 31, 2001
    Role: Principal Investigator
    CYTOTOXIC T CELLS AND MDX DYSTROPHY
    NIH/NIAMS F32AR008439Dec 20, 1996
    Role: Principal Investigator

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    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. to make corrections and additions.
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    Altmetrics Details 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. A Human Skeletal Muscle Atlas Identifies the Trajectories of Stem and Progenitor Cells across Development and from Human Pluripotent Stem Cells. Cell Stem Cell. 2020 Jul 02; 27(1):158-176.e10. Xi H, Langerman J, Sabri S, Chien P, Young CS, Younesi S, Hicks M, Gonzalez K, Fujiwara W, Marzi J, Liebscher S, Spencer M, Van Handel B, Evseenko D, Schenke-Layland K, Plath K, Pyle AD. PMID: 32396864.
      View in: PubMed   Mentions:    Fields:    
    2. Spp1 (osteopontin) promotes TGFß processing in fibroblasts of dystrophin-deficient muscles through matrix metalloproteinases. Hum Mol Genet. 2019 Oct 15; 28(20):3431-3442. Kramerova I, Kumagai-Cresse C, Ermolova N, Mokhonova E, Marinov M, Capote J, Becerra D, Quattrocelli M, Crosbie RH, Welch E, McNally EM, Spencer MJ. PMID: 31411676.
      View in: PubMed   Mentions: 2     Fields:    Translation:AnimalsCells
    3. The molecular tweezer CLR01 inhibits aberrant superoxide dismutase 1 (SOD1) self-assembly in vitro and in the G93A-SOD1 mouse model of ALS. J Biol Chem. 2019 03 08; 294(10):3501-3513. Malik R, Meng H, Wongkongkathep P, Corrales CI, Sepanj N, Atlasi RS, Klärner FG, Schrader T, Spencer MJ, Loo JA, Wiedau M, Bitan G. PMID: 30602569.
      View in: PubMed   Mentions: 4     Fields:    Translation:AnimalsCells
    4. Development of self-assembled multi-arm polyrotaxanes nanocarriers for systemic plasmid delivery in vivo. Biomaterials. 2019 02; 192:416-428. Ji Y, Liu X, Huang M, Jiang J, Liao YP, Liu Q, Chang CH, Liao H, Lu J, Wang X, Spencer MJ, Meng H. PMID: 30500723.
      View in: PubMed   Mentions: 1     Fields:    Translation:AnimalsCells
    5. Calpain 3 and CaMKIIß signaling are required to induce HSP70 necessary for adaptive muscle growth after atrophy. Hum Mol Genet. 2018 05 01; 27(9):1642-1653. Kramerova I, Torres JA, Eskin A, Nelson SF, Spencer MJ. PMID: 29528394.
      View in: PubMed   Mentions: 1     Fields:    Translation:AnimalsCells
    6. Repurposing Dantrolene for Long-Term Combination Therapy to Potentiate Antisense-Mediated DMD Exon Skipping in the mdx Mouse. Mol Ther Nucleic Acids. 2018 Jun 01; 11:180-191. Wang DW, Mokhonova EI, Kendall GC, Becerra D, Naeini YB, Cantor RM, Spencer MJ, Nelson SF, Miceli MC. PMID: 29858053.
      View in: PubMed   Mentions:
    7. ERBB3 and NGFR mark a distinct skeletal muscle progenitor cell in human development and hPSCs. Nat Cell Biol. 2018 01; 20(1):46-57. Hicks MR, Hiserodt J, Paras K, Fujiwara W, Eskin A, Jan M, Xi H, Young CS, Evseenko D, Nelson SF, Spencer MJ, Handel BV, Pyle AD. PMID: 29255171.
      View in: PubMed   Mentions: 23     Fields:    Translation:HumansCells
    8. Genetic modifiers of muscular dystrophy act on sarcolemmal resealing and recovery from injury. PLoS Genet. 2017 Oct; 13(10):e1007070. Quattrocelli M, Capote J, Ohiri JC, Warner JL, Vo AH, Earley JU, Hadhazy M, Demonbreun AR, Spencer MJ, McNally EM. PMID: 29065150.
      View in: PubMed   Mentions: 4     Fields:    Translation:AnimalsCells
    9. Vascular endothelium plays a key role in directing pulmonary epithelial cell differentiation. J Cell Biol. 2017 10 02; 216(10):3369-3385. Yao J, Guihard PJ, Wu X, Blazquez-Medela AM, Spencer MJ, Jumabay M, Tontonoz P, Fogelman AM, Boström KI, Yao Y. PMID: 28838957.
      View in: PubMed   Mentions: 4     Fields:    Translation:AnimalsCells
    10. Creation of a Novel Humanized Dystrophic Mouse Model of Duchenne Muscular Dystrophy and Application of a CRISPR/Cas9 Gene Editing Therapy. J Neuromuscul Dis. 2017; 4(2):139-145. Young CS, Mokhonova E, Quinonez M, Pyle AD, Spencer MJ. PMID: 28505980.
      View in: PubMed   Mentions: 12     Fields:    Translation:HumansAnimalsCells
    11. Outside in: The matrix as a modifier of muscular dystrophy. Biochim Biophys Acta Mol Cell Res. 2017 Mar; 1864(3):572-579. Quattrocelli M, Spencer MJ, McNally EM. PMID: 28011285.
      View in: PubMed   Mentions: 10     Fields:    Translation:HumansAnimalsCells
    12. High levels of sarcospan are well tolerated and act as a sarcolemmal stabilizer to address skeletal muscle and pulmonary dysfunction in DMD. Hum Mol Genet. 2016 12 15; 25(24):5395-5406. Gibbs EM, Marshall JL, Ma E, Nguyen TM, Hong G, Lam JS, Spencer MJ, Crosbie-Watson RH. PMID: 27798107.
      View in: PubMed   Mentions: 6     Fields:    Translation:HumansAnimalsCells
    13. Osteopontin ablation ameliorates muscular dystrophy by shifting macrophages to a pro-regenerative phenotype. J Cell Biol. 2016 04 25; 213(2):275-88. Capote J, Kramerova I, Martinez L, Vetrone S, Barton ER, Sweeney HL, Miceli MC, Spencer MJ. PMID: 27091452.
      View in: PubMed   Mentions: 28     Fields:    Translation:AnimalsCells
    14. Failure to up-regulate transcription of genes necessary for muscle adaptation underlies limb girdle muscular dystrophy 2A (calpainopathy). Hum Mol Genet. 2016 06 01; 25(11):2194-2207. Kramerova I, Ermolova N, Eskin A, Hevener A, Quehenberger O, Armando AM, Haller R, Romain N, Nelson SF, Spencer MJ. PMID: 27005420.
      View in: PubMed   Mentions: 8     Fields:    Translation:HumansAnimalsCells
    15. Attenuated Ca(2+) release in a mouse model of limb girdle muscular dystrophy 2A. Skelet Muscle. 2016; 6:11. DiFranco M, Kramerova I, Vergara JL, Spencer MJ. PMID: 26913171.
      View in: PubMed   Mentions: 4     Fields:    Translation:AnimalsCells
    16. A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle Cells. Cell Stem Cell. 2016 Apr 07; 18(4):533-40. Young CS, Hicks MR, Ermolova NV, Nakano H, Jan M, Younesi S, Karumbayaram S, Kumagai-Cresse C, Wang D, Zack JA, Kohn DB, Nakano A, Nelson SF, Miceli MC, Spencer MJ, Pyle AD. PMID: 26877224.
      View in: PubMed   Mentions: 87     Fields:    Translation:HumansAnimalsCells
    17. A reporter mouse for optical imaging of inflammation in mdx muscles. Skelet Muscle. 2015; 5:15. Martinez L, Ermolova NV, Ishikawa TO, Stout DB, Herschman HR, Spencer MJ. PMID: 25949789.
      View in: PubMed   Mentions: 2     Fields:    
    18. The E3 ubiquitin ligase TRIM32 regulates myoblast proliferation by controlling turnover of NDRG2. Hum Mol Genet. 2015 May 15; 24(10):2873-83. Mokhonova EI, Avliyakulov NK, Kramerova I, Kudryashova E, Haykinson MJ, Spencer MJ. PMID: 25701873.
      View in: PubMed   Mentions: 11     Fields:    Translation:AnimalsCells
    19. Autolytic activation of calpain 3 proteinase is facilitated by calmodulin protein. J Biol Chem. 2015 Jan 09; 290(2):996-1004. Ermolova N, Kramerova I, Spencer MJ. PMID: 25389288.
      View in: PubMed   Mentions: 4     Fields:    Translation:HumansAnimalsCells
    20. Regulatory T cells suppress muscle inflammation and injury in muscular dystrophy. Sci Transl Med. 2014 Oct 15; 6(258):258ra142. Villalta SA, Rosenthal W, Martinez L, Kaur A, Sparwasser T, Tidball JG, Margeta M, Spencer MJ, Bluestone JA. PMID: 25320234.
      View in: PubMed   Mentions: 65     Fields:    Translation:HumansAnimalsCells
    21. Dantrolene enhances antisense-mediated exon skipping in human and mouse models of Duchenne muscular dystrophy. Sci Transl Med. 2012 Dec 12; 4(164):164ra160. Kendall GC, Mokhonova EI, Moran M, Sejbuk NE, Wang DW, Silva O, Wang RT, Martinez L, Lu QL, Damoiseaux R, Spencer MJ, Nelson SF, Miceli MC. PMID: 23241744.
      View in: PubMed   Mentions: 33     Fields:    Translation:HumansAnimalsCells
    22. Skeletal muscle Nur77 expression enhances oxidative metabolism and substrate utilization. J Lipid Res. 2012 Dec; 53(12):2610-9. Chao LC, Wroblewski K, Ilkayeva OR, Stevens RD, Bain J, Meyer GA, Schenk S, Martinez L, Vergnes L, Narkar VA, Drew BG, Hong C, Boyadjian R, Hevener AL, Evans RM, Reue K, Spencer MJ, Newgard CB, Tontonoz P. PMID: 23028113.
      View in: PubMed   Mentions: 22     Fields:    Translation:AnimalsCells
    23. C3KO mouse expression analysis: downregulation of the muscular dystrophy Ky protein and alterations in muscle aging. Neurogenetics. 2012 Nov; 13(4):347-57. Jaka O, Kramerova I, Azpitarte M, López de Munain A, Spencer M, Sáenz A. PMID: 22820870.
      View in: PubMed   Mentions: 4     Fields:    Translation:HumansAnimals
    24. Satellite cell senescence underlies myopathy in a mouse model of limb-girdle muscular dystrophy 2H. J Clin Invest. 2012 May; 122(5):1764-76. Kudryashova E, Kramerova I, Spencer MJ. PMID: 22505452.
      View in: PubMed   Mentions: 33     Fields:    Translation:AnimalsCells
    25. The common missense mutation D489N in TRIM32 causing limb girdle muscular dystrophy 2H leads to loss of the mutated protein in knock-in mice resulting in a Trim32-null phenotype. Hum Mol Genet. 2011 Oct 15; 20(20):3925-32. Kudryashova E, Struyk A, Mokhonova E, Cannon SC, Spencer MJ. PMID: 21775502.
      View in: PubMed   Mentions: 19     Fields:    Translation:Animals
    26. Pathogenity of some limb girdle muscular dystrophy mutations can result from reduced anchorage to myofibrils and altered stability of calpain 3. Hum Mol Genet. 2011 Sep 01; 20(17):3331-45. Ermolova N, Kudryashova E, DiFranco M, Vergara J, Kramerova I, Spencer MJ. PMID: 21624972.
      View in: PubMed   Mentions: 12     Fields:    Translation:AnimalsCells
    27. Myogenic Akt signaling attenuates muscular degeneration, promotes myofiber regeneration and improves muscle function in dystrophin-deficient mdx mice. Hum Mol Genet. 2011 Apr 01; 20(7):1324-38. Kim MH, Kay DI, Rudra RT, Chen BM, Hsu N, Izumiya Y, Martinez L, Spencer MJ, Walsh K, Grinnell AD, Crosbie RH. PMID: 21245083.
      View in: PubMed   Mentions: 17     Fields:    Translation:HumansAnimalsCells
    28. Limb-girdle muscular dystrophy 2H and the role of TRIM32. Handb Clin Neurol. 2011; 101:125-33. Shieh PB, Kudryashova E, Spencer MJ. PMID: 21496629.
      View in: PubMed   Mentions: 9     Fields:    Translation:Humans
    29. Emerging genetic therapies to treat Duchenne muscular dystrophy. Curr Opin Neurol. 2009 Oct; 22(5):532-8. Nelson SF, Crosbie RH, Miceli MC, Spencer MJ. PMID: 19745732.
      View in: PubMed   Mentions: 23     Fields:    Translation:Humans
    30. Calcium-dependent plasma membrane repair requires m- or mu-calpain, but not calpain-3, the proteasome, or caspases. Biochim Biophys Acta. 2009 Dec; 1793(12):1886-93. Mellgren RL, Miyake K, Kramerova I, Spencer MJ, Bourg N, Bartoli M, Richard I, Greer PA, McNeil PL. PMID: 19781581.
      View in: PubMed   Mentions: 29     Fields:    Translation:AnimalsCells
    31. Mitochondrial abnormalities, energy deficit and oxidative stress are features of calpain 3 deficiency in skeletal muscle. Hum Mol Genet. 2009 Sep 01; 18(17):3194-205. Kramerova I, Kudryashova E, Wu B, Germain S, Vandenborne K, Romain N, Haller RG, Verity MA, Spencer MJ. PMID: 19483197.
      View in: PubMed   Mentions: 19     Fields:    Translation:AnimalsCells
    32. Osteopontin promotes fibrosis in dystrophic mouse muscle by modulating immune cell subsets and intramuscular TGF-beta. J Clin Invest. 2009 Jun; 119(6):1583-94. Vetrone SA, Montecino-Rodriguez E, Kudryashova E, Kramerova I, Hoffman EP, Liu SD, Miceli MC, Spencer MJ. PMID: 19451692.
      View in: PubMed   Mentions: 107     Fields:    Translation:HumansAnimalsCells
    33. Deficiency of the E3 ubiquitin ligase TRIM32 in mice leads to a myopathy with a neurogenic component. Hum Mol Genet. 2009 Apr 01; 18(7):1353-67. Kudryashova E, Wu J, Havton LA, Spencer MJ. PMID: 19155210.
      View in: PubMed   Mentions: 42     Fields:    Translation:AnimalsCells
    34. Calpain 3, the "gatekeeper" of proper sarcomere assembly, turnover and maintenance. Neuromuscul Disord. 2008 Dec; 18(12):913-21. Beckmann JS, Spencer M. PMID: 18974005.
      View in: PubMed   Mentions: 41     Fields:    Translation:HumansAnimalsCells
    35. Novel role of calpain-3 in the triad-associated protein complex regulating calcium release in skeletal muscle. Hum Mol Genet. 2008 Nov 01; 17(21):3271-80. Kramerova I, Kudryashova E, Wu B, Ottenheijm C, Granzier H, Spencer MJ. PMID: 18676612.
      View in: PubMed   Mentions: 34     Fields:    Translation:HumansAnimalsCells
    36. Calpain activation impairs neuromuscular transmission in a mouse model of the slow-channel myasthenic syndrome. J Clin Invest. 2007 Oct; 117(10):2903-12. Groshong JS, Spencer MJ, Bhattacharyya BJ, Kudryashova E, Vohra BP, Zayas R, Wollmann RL, Miller RJ, Gomez CM. PMID: 17853947.
      View in: PubMed   Mentions: 15     Fields:    Translation:HumansAnimalsCells
    37. Identification of putative in vivo substrates of calpain 3 by comparative proteomics of overexpressing transgenic and nontransgenic mice. Proteomics. 2006 Nov; 6(22):6075-84. Cohen N, Kudryashova E, Kramerova I, Anderson LV, Beckmann JS, Bushby K, Spencer MJ. PMID: 17051641.
      View in: PubMed   Mentions: 14     Fields:    Translation:Animals
    38. Regulation of the M-cadherin-beta-catenin complex by calpain 3 during terminal stages of myogenic differentiation. Mol Cell Biol. 2006 Nov; 26(22):8437-47. Kramerova I, Kudryashova E, Wu B, Spencer MJ. PMID: 16982691.
      View in: PubMed   Mentions: 24     Fields:    Translation:AnimalsCells
    39. Molecular and cellular basis of calpainopathy (limb girdle muscular dystrophy type 2A). Biochim Biophys Acta. 2007 Feb; 1772(2):128-44. Kramerova I, Beckmann JS, Spencer MJ. PMID: 16934440.
      View in: PubMed   Mentions: 23     Fields:    Translation:HumansAnimals
    40. Reinvestigation of the dysbindin subunit of BLOC-1 (biogenesis of lysosome-related organelles complex-1) as a dystrobrevin-binding protein. Biochem J. 2006 May 01; 395(3):587-98. Nazarian R, Starcevic M, Spencer MJ, Dell'Angelica EC. PMID: 16448387.
      View in: PubMed   Mentions: 32     Fields:    Translation:HumansAnimalsCells
    41. Trim32 is a ubiquitin ligase mutated in limb girdle muscular dystrophy type 2H that binds to skeletal muscle myosin and ubiquitinates actin. J Mol Biol. 2005 Nov 25; 354(2):413-24. Kudryashova E, Kudryashov D, Kramerova I, Spencer MJ. PMID: 16243356.
      View in: PubMed   Mentions: 70     Fields:    Translation:AnimalsCells
    42. Mdm muscular dystrophy: interactions with calpain 3 and a novel functional role for titin's N2A domain. Hum Mol Genet. 2005 Oct 01; 14(19):2801-11. Huebsch KA, Kudryashova E, Wooley CM, Sher RB, Seburn KL, Spencer MJ, Cox GA. PMID: 16115818.
      View in: PubMed   Mentions: 16     Fields:    Translation:AnimalsCells
    43. Calpain 3 participates in sarcomere remodeling by acting upstream of the ubiquitin-proteasome pathway. Hum Mol Genet. 2005 Aug 01; 14(15):2125-34. Kramerova I, Kudryashova E, Venkatraman G, Spencer MJ. PMID: 15961411.
      View in: PubMed   Mentions: 48     Fields:    Translation:AnimalsCells
    44. Null mutation of calpain 3 (p94) in mice causes abnormal sarcomere formation in vivo and in vitro. Hum Mol Genet. 2004 Jul 01; 13(13):1373-88. Kramerova I, Kudryashova E, Tidball JG, Spencer MJ. PMID: 15138196.
      View in: PubMed   Mentions: 63     Fields:    Translation:AnimalsCells
    45. Calpain 3 cleaves filamin C and regulates its ability to interact with gamma- and delta-sarcoglycans. Muscle Nerve. 2003 Oct; 28(4):472-83. Guyon JR, Kudryashova E, Potts A, Dalkilic I, Brosius MA, Thompson TG, Beckmann JS, Kunkel LM, Spencer MJ. PMID: 14506720.
      View in: PubMed   Mentions: 27     Fields:    Translation:AnimalsCells
    46. Ventilatory dysfunction in mdx mice: impact of tumor necrosis factor-alpha deletion. Muscle Nerve. 2003 Sep; 28(3):336-43. Gosselin LE, Barkley JE, Spencer MJ, McCormick KM, Farkas GA. PMID: 12929194.
      View in: PubMed   Mentions: 20     Fields:    Translation:AnimalsCells
    47. Skipping to new gene therapies for muscular dystrophy. Nat Med. 2003 Aug; 9(8):997-8. Tidball JG, Spencer MJ. PMID: 12894160.
      View in: PubMed   Mentions: 4     Fields:    Translation:HumansAnimalsCells
    48. A role for natural killer cells in the rapid death of cultured donor myoblasts after transplantation. Transplantation. 2003 Mar 27; 75(6):863-71. Hodgetts SI, Spencer MJ, Grounds MD. PMID: 12660516.
      View in: PubMed   Mentions: 3     Fields:    Translation:AnimalsCells
    49. Expression of a calpastatin transgene slows muscle wasting and obviates changes in myosin isoform expression during murine muscle disuse. J Physiol. 2002 12 15; 545(3):819-28. Tidball JG, Spencer MJ. PMID: 12482888.
      View in: PubMed   Mentions: 58     Fields:    Translation:HumansAnimalsCells
    50. Overexpression of a calpastatin transgene in mdx muscle reduces dystrophic pathology. Hum Mol Genet. 2002 Oct 01; 11(21):2645-55. Spencer MJ, Mellgren RL. PMID: 12354790.
      View in: PubMed   Mentions: 52     Fields:    Translation:AnimalsCells
    51. Stable expression of calpain 3 from a muscle transgene in vivo: immature muscle in transgenic mice suggests a role for calpain 3 in muscle maturation. Proc Natl Acad Sci U S A. 2002 Jun 25; 99(13):8874-9. Spencer MJ, Guyon JR, Sorimachi H, Potts A, Richard I, Herasse M, Chamberlain J, Dalkilic I, Kunkel LM, Beckmann JS. PMID: 12084932.
      View in: PubMed   Mentions: 30     Fields:    Translation:HumansAnimalsCells
    52. Quantification of splice variants using molecular beacon or scorpion primers. Anal Biochem. 2002 Jun 15; 305(2):227-35. Taveau M, Stockholm D, Spencer M, Richard I. PMID: 12054451.
      View in: PubMed   Mentions: 3     Fields:    Translation:AnimalsCells
    53. Do immune cells promote the pathology of dystrophin-deficient myopathies? Neuromuscul Disord. 2001 Sep; 11(6-7):556-64. Spencer MJ, Tidball JG. PMID: 11525885.
      View in: PubMed   Mentions: 56     Fields:    Translation:HumansAnimalsCells
    54. Altered pathological progression of diaphragm and quadriceps muscle in TNF-deficient, dystrophin-deficient mice. Neuromuscul Disord. 2000 Dec; 10(8):612-9. Spencer MJ, Marino MW, Winckler WM. PMID: 11053690.
      View in: PubMed   Mentions: 15     Fields:    Translation:AnimalsCells
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