An mTOR Signaling Modulator Suppressed Heterotopic Ossification of Fibrodysplasia Ossificans Progressiva

[1]  J. Toguchida,et al.  Modeling human somite development and fibrodysplasia ossificans progressiva with induced pluripotent stem cells , 2018, Development.

[2]  V. Kaartinen,et al.  Activin-dependent signaling in fibro/adipogenic progenitors causes fibrodysplasia ossificans progressiva , 2018, Nature Communications.

[3]  T. Kambayashi,et al.  Depletion of Mast Cells and Macrophages Impairs Heterotopic Ossification in an Acvr1R206H Mouse Model of Fibrodysplasia Ossificans Progressiva , 2018, Journal of bone and mineral research : the official journal of the American Society for Bone and Mineral Research.

[4]  R. Pignolo,et al.  Mast cell inhibition as a therapeutic approach in fibrodysplasia ossificans progressiva (FOP). , 2017, Bone.

[5]  K. Kawakami,et al.  Activin-A enhances mTOR signaling to promote aberrant chondrogenesis in fibrodysplasia ossificans progressiva. , 2017, The Journal of clinical investigation.

[6]  M. Prunotto,et al.  Opportunities and challenges in phenotypic drug discovery: an industry perspective , 2017, Nature Reviews Drug Discovery.

[7]  V. Kaartinen,et al.  Scleraxis‐Lineage Cells Contribute to Ectopic Bone Formation in Muscle and Tendon , 2017, Stem cells.

[8]  S. Stagi,et al.  Bone modeling, remodeling, and skeletal health in children and adolescents: mineral accrual, assessment and treatment , 2017, Annals of pediatric endocrinology & metabolism.

[9]  C. Keller,et al.  Two tissue-resident progenitor lineages drive distinct phenotypes of heterotopic ossification , 2016, Science Translational Medicine.

[10]  A. Economides,et al.  Palovarotene Inhibits Heterotopic Ossification and Maintains Limb Mobility and Growth in Mice With the Human ACVR1R206H Fibrodysplasia Ossificans Progressiva (FOP) Mutation , 2016, Journal of bone and mineral research : the official journal of the American Society for Bone and Mineral Research.

[11]  R. Pignolo,et al.  Cellular Hypoxia Promotes Heterotopic Ossification by Amplifying BMP Signaling , 2016, Journal of bone and mineral research : the official journal of the American Society for Bone and Mineral Research.

[12]  Cary L. Honnold,et al.  Targeted stimulation of retinoic acid receptor-γ mitigates the formation of heterotopic ossification in an established blast-related traumatic injury model. , 2016, Bone.

[13]  Y. Usami,et al.  Effectiveness and mode of action of a combination therapy for heterotopic ossification with a retinoid agonist and an anti-inflammatory agent. , 2016, Bone.

[14]  R. Ravazzolo,et al.  High-throughput screening for modulators of ACVR1 transcription: discovery of potential therapeutics for fibrodysplasia ossificans progressiva , 2016, Disease Models & Mechanisms.

[15]  David M. Rocke,et al.  The Natural History of Flare‐Ups in Fibrodysplasia Ossificans Progressiva (FOP): A Comprehensive Global Assessment , 2016, Journal of bone and mineral research : the official journal of the American Society for Bone and Mineral Research.

[16]  R. Tompkins,et al.  Inhibition of Hif1α prevents both trauma-induced and genetic heterotopic ossification , 2015, Proceedings of the National Academy of Sciences.

[17]  Y. Matsumoto,et al.  Neofunction of ACVR1 in fibrodysplasia ossificans progressiva , 2015, Proceedings of the National Academy of Sciences.

[18]  Lily Huang,et al.  ACVR1R206H receptor mutation causes fibrodysplasia ossificans progressiva by imparting responsiveness to activin A , 2015, Science Translational Medicine.

[19]  Y. Matsumoto,et al.  New Protocol to Optimize iPS Cells for Genome Analysis of Fibrodysplasia Ossificans Progressiva , 2015, Stem cells.

[20]  Y. Matsumoto,et al.  Derivation of Mesenchymal Stromal Cells from Pluripotent Stem Cells through a Neural Crest Lineage using Small Molecule Compounds with Defined Media , 2014, PloS one.

[21]  S. Yamanaka,et al.  Premature Termination of Reprogramming In Vivo Leads to Cancer Development through Altered Epigenetic Regulation , 2014, Cell.

[22]  B. Conklin,et al.  Induced pluripotent stem cells from patients with human fibrodysplasia ossificans progressiva show increased mineralization and cartilage formation , 2013, Orphanet Journal of Rare Diseases.

[23]  K. Ohno,et al.  Perhexiline maleate in the treatment of fibrodysplasia ossificans progressiva: an open-labeled clinical trial , 2013, Orphanet Journal of Rare Diseases.

[24]  Ron Prywes,et al.  Serum regulation of Id1 expression by a BMP pathway and BMP responsive element. , 2013, Biochimica et biophysica acta.

[25]  A. Bullock,et al.  Development of an ALK2-biased BMP type I receptor kinase inhibitor. , 2013, ACS chemical biology.

[26]  Charles C Hong,et al.  Synthesis and structure-activity relationships of a novel and selective bone morphogenetic protein receptor (BMP) inhibitor derived from the pyrazolo[1.5-a]pyrimidine scaffold of dorsomorphin: the discovery of ML347 as an ALK2 versus ALK3 selective MLPCN probe. , 2013, Bioorganic & medicinal chemistry letters.

[27]  A. Bullock,et al.  A New Class of Small Molecule Inhibitor of BMP Signaling , 2013, PloS one.

[28]  K. Woltjen,et al.  Identification of target genes of synovial sarcoma-associated fusion oncoprotein using human pluripotent stem cells. , 2013, Biochemical and biophysical research communications.

[29]  A. Hara,et al.  EWS/ATF1 expression induces sarcomas from neural crest-derived cells in mice. , 2013, The Journal of clinical investigation.

[30]  Y. Matsumoto,et al.  Genetically Matched Human iPS Cells Reveal that Propensity for Cartilage and Bone Differentiation Differs with Clones, not Cell Type of Origin , 2013, PloS one.

[31]  F. Kaplan,et al.  Fibrodysplasia ossificans progressiva: mechanisms and models of skeletal metamorphosis , 2012, Disease Models & Mechanisms.

[32]  M. Hamasaki,et al.  Pathogenic Mutation of ALK2 Inhibits Induced Pluripotent Stem Cell Reprogramming and Maintenance: Mechanisms of Reprogramming and Strategy for Drug Identification , 2012, Stem cells.

[33]  Stefan Knapp,et al.  Structure of the Bone Morphogenetic Protein Receptor ALK2 and Implications for Fibrodysplasia Ossificans Progressiva , 2012, The Journal of Biological Chemistry.

[34]  J. Nickel,et al.  Promiscuity and specificity in BMP receptor activation , 2012, FEBS letters.

[35]  E. Stanley,et al.  Human chondrogenic paraxial mesoderm, directed specification and prospective isolation from pluripotent stem cells , 2012, Scientific Reports.

[36]  H. Hohjoh,et al.  Disease-causing allele-specific silencing against the ALK2 mutants, R206H and G356D, in fibrodysplasia ossificans progressiva , 2011, Gene Therapy.

[37]  E. Shore,et al.  Restoration of normal BMP signaling levels and osteogenic differentiation in FOP mesenchymal progenitor cells by mutant allele-specific targeting , 2011, Gene Therapy.

[38]  Theonie Anastassiadis,et al.  Comprehensive assay of kinase catalytic activity reveals features of kinase inhibitor selectivity , 2011, Nature Biotechnology.

[39]  Masahiro Iwamoto,et al.  Potent Inhibition of Heterotopic Ossification by Nuclear Retinoic Acid Receptor γ Agonists , 2011, Nature Medicine.

[40]  F. Kaplan,et al.  Inherited human diseases of heterotopic bone formation , 2010, Nature Reviews Rheumatology.

[41]  C. Lindsley,et al.  In vivo structure-activity relationship study of dorsomorphin analogues identifies selective VEGF and BMP inhibitors. , 2010, ACS chemical biology.

[42]  G. Volpin,et al.  Treatment of articular cartilage lesions of the knee , 2010, International Orthopaedics.

[43]  Wei Wang,et al.  piggyBac transposition reprograms fibroblasts to induced pluripotent stem cells , 2009, Nature.

[44]  Y. Okazaki,et al.  A unique mutation of ALK2, G356D, found in a patient with fibrodysplasia ossificans progressiva is a moderately activated BMP type I receptor. , 2008, Biochemical and biophysical research communications.

[45]  Tomokazu Fukuda,et al.  BMP type I receptor inhibition reduces heterotopic ossification , 2008, Nature Medicine.

[46]  F. Kaplan,et al.  Fibrodysplasia ossificans progressiva , 2008, Best practice & research. Clinical rheumatology.

[47]  R. O’Keefe,et al.  Regulation of chondrogenesis and chondrocyte differentiation by stress. , 2008, The Journal of clinical investigation.

[48]  R. Pignolo,et al.  Morphogen Receptor Genes and Metamorphogenes , 2007 .

[49]  P. Billings,et al.  Dysregulated BMP Signaling and Enhanced Osteogenic Differentiation of Connective Tissue Progenitor Cells From Patients With Fibrodysplasia Ossificans Progressiva (FOP) , 2007, Journal of bone and mineral research : the official journal of the American Society for Bone and Mineral Research.

[50]  M. Moasser Targeting the function of the HER2 oncogene in human cancer therapeutics , 2007, Oncogene.

[51]  L. Otterbein,et al.  N-(5-chloro-1,3-benzodioxol-4-yl)-7-[2-(4-methylpiperazin-1-yl)ethoxy]-5- (tetrahydro-2H-pyran-4-yloxy)quinazolin-4-amine, a novel, highly selective, orally available, dual-specific c-Src/Abl kinase inhibitor. , 2006, Journal of medicinal chemistry.

[52]  Mikio Namiki,et al.  Novel HER2 selective tyrosine kinase inhibitor, TAK‐165, inhibits bladder, kidney and androgen‐independent prostate cancer in vitro and in vivo , 2006, International journal of urology : official journal of the Japanese Urological Association.

[53]  Rudolf Jaenisch,et al.  Efficient method to generate single‐copy transgenic mice by site‐specific integration in embryonic stem cells , 2006, Genesis.

[54]  J. Connor,et al.  The phenotype of fibrodysplasia ossificans progressiva , 2005 .

[55]  F. Kaplan,et al.  The genetics of fibrodysplasia ossificans progressiva , 2005 .

[56]  A. Economides,et al.  0163-769X/03/$20.00/0 Endocrine Reviews 24(2):218–235 Printed in U.S.A. Copyright © 2003 by The Endocrine Society doi: 10.1210/er.2002-0023 Bone Morphogenetic Proteins, Their Antagonists, and the Skeleton , 2022 .

[57]  J. Massagué,et al.  TGFβ Signaling in Growth Control, Cancer, and Heritable Disorders , 2000, Cell.

[58]  T. Nakamura,et al.  Differential expressions of BMP family genes during chondrogenic differentiation of mouse ATDC5 cells. , 2000, Cell structure and function.

[59]  C. Heldin,et al.  Specificity, diversity, and regulation in TGF‐β superfamily signaling , 1999 .

[60]  A. Bradley,et al.  Multiple roles for activin-like kinase-2 signaling during mouse embryogenesis. , 1999, Developmental biology.

[61]  P. Donahoe,et al.  The type I serine/threonine kinase receptor ActRIA (ALK2) is required for gastrulation of the mouse embryo. , 1999, Development.

[62]  T. Atsumi,et al.  Cellular Hypertrophy and Calcification of Embryonal Carcinoma‐Derived Chondrogenic Cell Line ATDC5 In Vitro , 1997, Journal of bone and mineral research : the official journal of the American Society for Bone and Mineral Research.

[63]  B. Hogan,et al.  Bone morphogenetic proteins: multifunctional regulators of vertebrate development. , 1996, Genes & development.

[64]  V. Rosen,et al.  Novel regulators of bone formation: molecular clones and activities. , 1988, Science.

[65]  M. Urist,et al.  Bone: Formation by Autoinduction , 1965, Science.

[66]  Kohei Miyazono,et al.  Bone morphogenetic protein receptors and signal transduction. , 2010, Journal of biochemistry.

[67]  In Ho Choi,et al.  A recurrent mutation in the BMP type I receptor ACVR1 causes inherited and sporadic fibrodysplasia ossificans progressiva , 2006, Nature Genetics.