1,801
Views
28
CrossRef citations to date
0
Altmetric
Editorial

Hard targets for a second skeleton: therapeutic horizons for fibrodysplasia ossificans progressiva (FOP)

, , &
Pages 291-294 | Received 06 Feb 2017, Accepted 06 Mar 2017, Published online: 17 Mar 2017

References

  • Kaplan FS, Pignolo RJ, Shore EM. From mysteries to medicines: drug development for fibrodysplasia ossificans progressive. Expert Opin Orphan Drugs. 2013;1:637–649.
  • Pignolo RJ, Bedford-Gay C, Liljesthrom M, et al. The natural history of flare-ups in fibrodysplasia ossificans progressiva: a comprehensive global assessment. J Bone Miner Res. 2016;31:650–656.
  • Shore EM, Xu M, Feldman GJ, et al. A recurrent mutation in the BMP type I receptor ACVR1 causes inherited and sporadic fibrodysplasia ossificans progressiva. Nat Genet. 2016;38:525–527.
  • Hatsell SJ, Idone V, Wolken DM, et al. ACVR1 (R206H) receptor mutation causes fibrodysplasia ossificans progressiva by imparting responsiveness to Activin A. Sci Transl Med. 2015;7(303):ra137.
  • Hino K, Ikeya M, Horigome K, et al. Neofunction of ACVR1 in fibrodysplasia ossificans progressiva. Proc Natl Acad Sci USA. 2015;112:15438–15443.
  • Kaplan FS, Shore EM, Pignolo RJ, eds; The International Clinical Consortium on FOP. The medical management of fibrodysplasia ossificans progressiva: current treatment considerations. Clin Proc Intl Clin Consort FOP. 2011;4:1–100. Available from: www.ifopa.org
  • Chakkalakal SA, Zhang D, Culbert AL, et al. An Acvr1 R206H knock-in mouse has fibrodysplasia ossificans progressiva. J Bone Miner Res. 2012;27:1746–1756.
  • Hong CC, Yu PB. Applications of small molecule BMP inhibitors in physiology and disease. Cytokine Growth Factor Rev. 2009;20:409–418.
  • Dey D, Bagarova J, Hatsell SJ, et al. Two tissue-resident progenitor lineages drive distinct phenotypes of heterotopic ossification. Sci Transl Med. 2016;8(366):366ra163.
  • Kaplan FS, Pignolo RJ, Shore EM. Granting immunity to FOP and catching heterotopic ossification in the Act. Semin Cell Dev Biol. 2016;49:30–36.
  • Pang J, Zuo Y, Chen Y, et al. ACVR1-Fc suppresses BMP signaling and chondro-osseous differentiation in an in vitro model of fibrodysplasia ossificans progressiva. Bone. 2016;92:29–36.
  • Lowery JW, Rosen V. Allele-specific RNA interference in FOP: silencing the FOP gene. Gene Ther. 2012;19:701–702.
  • Kan L, Lounev VY, Pignolo RJ, et al. Substance P signaling mediates BMP-dependent heterotopic ossification. J Cell Biochem. 2011;112:2759–2772.
  • Kaplan FS, Glaser DL, Shore EM, et al. Hematopoietic stem-cell contribution to ectopic skeletogenesis. J Bone Joint Surg Am. 2007;89:347–357.
  • Shimono K, Tung WE, Macolino C, et al. Potent inhibition of heterotopic ossification by nuclear retinoic acid receptor-gamma agonists. Nat Med. 2011;17:454–460.
  • Chakkalakal SA, Uchibe K, Convente MR, et al. Palovarotene inhibits heterotopic ossification and maintains limb mobility and growth in mice with the human ACVR1 (R206H) fibrodysplasia ossificans progressiva (FOP) mutation. J Bone Miner Res. 2016;31:1666–1675.
  • Wang H, Lindborg C, Lounev V, et al. Cellular hypoxia promotes heterotopic ossification by amplifying BMP signaling. J Bone Miner Res. 2016;31:1652–1665.
  • Agarwal S, Loder S, Brownley C, et al. Inhibition of Hif1α prevents both trauma-induced and genetic heterotopic ossification. Proc Natl Acad Sci USA. 2016;113:E338–347.

Reprints and Corporate Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

To request a reprint or corporate permissions for this article, please click on the relevant link below:

Academic Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

Obtain permissions instantly via Rightslink by clicking on the button below:

If you are unable to obtain permissions via Rightslink, please complete and submit this Permissions form. For more information, please visit our Permissions help page.