Literature DB >> 25337067

Fibrodysplasia ossificans progressiva: clinical course, genetic mutations and genotype-phenotype correlation.

Irina Hüning1, Gabriele Gillessen-Kaesbach1.   

Abstract

Fibrodysplasia ossificans progressiva (FOP, MIM 135100) is a rare autosomal dominant genetic disorder and the most disabling condition of heterotopic (extraskeletal) ossification in humans. Mutations in the ACVR1 gene (MIM 102576) were identified as a genetic cause of FOP [Shore et al., 2006]. Most patients with FOP have the same recurrent single nucleotide change c.617G>A, p.R206H in the ACVR1 gene. Furthermore, 11 other mutations in the ACVR1 gene have been described as a cause of FOP. Here, we review phenotypic and molecular findings of 130 cases of FOP reported in the literature from 1982 to April 2014 and discuss possible genotype-phenotype correlations in FOP patients.

Entities:  

Keywords:  ACVR1; FOP; Great toe malformations; Heterotopic ossifications; Progressive immobility

Year:  2014        PMID: 25337067      PMCID: PMC4188166          DOI: 10.1159/000365770

Source DB:  PubMed          Journal:  Mol Syndromol        ISSN: 1661-8769


  35 in total

Review 1.  Mechanisms of TGF-beta signaling from cell membrane to the nucleus.

Authors:  Yigong Shi; Joan Massagué
Journal:  Cell       Date:  2003-06-13       Impact factor: 41.582

Review 2.  Smad transcription factors.

Authors:  Joan Massagué; Joan Seoane; David Wotton
Journal:  Genes Dev       Date:  2005-12-01       Impact factor: 11.361

3.  A novel ACVR1 mutation in the glycine/serine-rich domain found in the most benign case of a fibrodysplasia ossificans progressiva variant reported to date.

Authors:  Celia L Gregson; Peter Hollingworth; Martin Williams; Kirsten A Petrie; Alex N Bullock; Matthew A Brown; Jon H Tobias; James T Triffitt
Journal:  Bone       Date:  2010-10-29       Impact factor: 4.398

4.  Bone: formation by autoinduction.

Authors:  M R Urist
Journal:  Science       Date:  1965-11-12       Impact factor: 47.728

5.  Conductive hearing loss in individuals with fibrodysplasia ossificans progressiva.

Authors:  C E Levy; A T Lash; H B Janoff; F S Kaplan
Journal:  Am J Audiol       Date:  1999-06       Impact factor: 1.493

6.  A unique case of fibrodysplasia ossificans progressiva with an ACVR1 mutation, G356D, other than the common mutation (R206H).

Authors:  Hirokazu Furuya; Koji Ikezoe; Lixiang Wang; Yasumasa Ohyagi; Kyoko Motomura; Naoki Fujii; Jun-Ichi Kira; Yasuyuki Fukumaki
Journal:  Am J Med Genet A       Date:  2008-02-15       Impact factor: 2.802

7.  Fibrodysplasia ossificans progressiva. The clinical features and natural history of 34 patients.

Authors:  J M Connor; D A Evans
Journal:  J Bone Joint Surg Br       Date:  1982

Review 8.  Bone morphogenetic protein signaling in stem cells--one signal, many consequences.

Authors:  Toni U Wagner
Journal:  FEBS J       Date:  2007-05-22       Impact factor: 5.542

9.  The face signature of fibrodysplasia ossificans progressiva.

Authors:  Peter Hammond; Michael Suttie; Raoul C Hennekam; Judith Allanson; Eileen M Shore; Frederick S Kaplan
Journal:  Am J Med Genet A       Date:  2012-05-11       Impact factor: 2.802

10.  Novel mutations in ACVR1 result in atypical features in two fibrodysplasia ossificans progressiva patients.

Authors:  Kirsten A Petrie; Wen Hwa Lee; Alex N Bullock; Jenny J Pointon; Roger Smith; R Graham G Russell; Matthew A Brown; B Paul Wordsworth; James T Triffitt
Journal:  PLoS One       Date:  2009-03-30       Impact factor: 3.240

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  28 in total

1.  Variant BMP receptor mutations causing fibrodysplasia ossificans progressiva (FOP) in humans show BMP ligand-independent receptor activation in zebrafish.

Authors:  Bettina E Mucha; Megumi Hashiguchi; Joseph Zinski; Eileen M Shore; Mary C Mullins
Journal:  Bone       Date:  2018-01-04       Impact factor: 4.398

2.  Primum non nocere: a case of a humeral fracture in a patient with fibrodysplasia progressiva ossificans.

Authors:  Alun Yewlett; Jeff Kitson; Andrew Redfern; Chris Smith
Journal:  Shoulder Elbow       Date:  2015-08-10

3.  Fibrodysplasia ossificans progressiva (FOP) presenting as a rapidly growing non-calcified neck mass.

Authors:  Ghiam Yamin; Shadi Daghighi; Mahmood Mafee
Journal:  J Radiol Case Rep       Date:  2021-05-31

4.  Myositis ossificans in children: a review.

Authors:  N K Sferopoulos; R Kotakidou; A S Petropoulos
Journal:  Eur J Orthop Surg Traumatol       Date:  2017-03-09

Review 5.  BMP signalling in skeletal development, disease and repair.

Authors:  Valerie S Salazar; Laura W Gamer; Vicki Rosen
Journal:  Nat Rev Endocrinol       Date:  2016-02-19       Impact factor: 43.330

6.  Clinical and molecular characterisation of two siblings with fibrodysplasia ossificans progressiva, from the Colombian Pacific coast (South America).

Authors:  Harry Pachajoa; Andres Felipe Ramirez Botero
Journal:  BMJ Case Rep       Date:  2015-06-08

7.  Multi-system involvement in a severe variant of fibrodysplasia ossificans progressiva (ACVR1 c.772G>A; R258G): A report of two patients.

Authors:  Frederick S Kaplan; Joyce A Kobori; Carmen Orellana; Inmaculada Calvo; Monica Rosello; Francisco Martinez; Berta Lopez; Meiqi Xu; Robert J Pignolo; Eileen M Shore; Jay C Groppe
Journal:  Am J Med Genet A       Date:  2015-06-11       Impact factor: 2.802

Review 8.  Bone Morphogenetic Protein-Based Therapeutic Approaches.

Authors:  Jonathan W Lowery; Vicki Rosen
Journal:  Cold Spring Harb Perspect Biol       Date:  2018-04-02       Impact factor: 10.005

9.  ACVR1R206H receptor mutation causes fibrodysplasia ossificans progressiva by imparting responsiveness to activin A.

Authors:  Sarah J Hatsell; Vincent Idone; Dana M Alessi Wolken; Lily Huang; Hyon J Kim; Lili Wang; Xialing Wen; Kalyan C Nannuru; Johanna Jimenez; Liqin Xie; Nanditha Das; Genevieve Makhoul; Rostislav Chernomorsky; David D'Ambrosio; Richard A Corpina; Christopher J Schoenherr; Kieran Feeley; Paul B Yu; George D Yancopoulos; Andrew J Murphy; Aris N Economides
Journal:  Sci Transl Med       Date:  2015-09-02       Impact factor: 17.956

Review 10.  Granting immunity to FOP and catching heterotopic ossification in the Act.

Authors:  Frederick S Kaplan; Robert J Pignolo; Eileen M Shore
Journal:  Semin Cell Dev Biol       Date:  2015-12-17       Impact factor: 7.727

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