Literature DB >> 22987702

Muscle function in a canine model of X-linked myotubular myopathy.

Robert W Grange1, Jon Doering, Erin Mitchell, Melanie N Holder, Xuan Guan, Melissa Goddard, Christopher Tegeler, Alan H Beggs, Martin K Childers.   

Abstract

INTRODUCTION: We established a colony of dogs that harbor an X-linked MTM1 missense mutation.Muscle from affected male dogs exhibits reduction and altered localization of the MTM1 gene product, myotubularin, and provides a model analogous to X-linked myotubular myopathy (XLMTM).
METHODS: We studied hindlimb muscle function in age-matched canine XLMTM genotypes between ages 9 and 18 weeks.
RESULTS: By the end of the study, affected dogs produce only ∼15% of the torque generated by normals or carriers (0.023 ± 0.005 vs. 0.152 ± 0.007 and 0.154 ± 0.003 N-m/kg body mass, respectively, P < 0.05) and are too weak to stand unassisted. At this age, XLMTM dogs also demonstrate an abnormally low twitch:tetanus ratio, a right-shifted torque-frequency relationship and an increase in torque during repetitive stimulation (P < 0.05).
CONCLUSIONS: We hypothesize that muscle weakness results from impaired excitation-contraction (E-C) coupling. Interventions that improve E-C coupling might be translated from the XLMTM dog model to patients.
Copyright © 2012 Wiley Periodicals, Inc.

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Year:  2012        PMID: 22987702      PMCID: PMC3448125          DOI: 10.1002/mus.23463

Source DB:  PubMed          Journal:  Muscle Nerve        ISSN: 0148-639X            Impact factor:   3.217


  16 in total

1.  The myotubularin family: from genetic disease to phosphoinositide metabolism.

Authors:  J Laporte; F Blondeau; A Buj-Bello; J L Mandel
Journal:  Trends Genet       Date:  2001-04       Impact factor: 11.639

2.  Prenatal diagnosis of X-linked myotubular myopathy: strategies using new and tightly linked DNA markers.

Authors:  L J Hu; J Laporte; W Kress; N Dahl
Journal:  Prenat Diagn       Date:  1996-03       Impact factor: 3.050

3.  Medical complications in long-term survivors with X-linked myotubular myopathy.

Authors:  G E Herman; M Finegold; W Zhao; B de Gouyon; A Metzenberg
Journal:  J Pediatr       Date:  1999-02       Impact factor: 4.406

4.  Congenital centronuclear (myotubular) myopathy. A clinical, pathological and genetic study in eight children.

Authors:  J Z Heckmatt; C A Sewry; D Hodes; V Dubowitz
Journal:  Brain       Date:  1985-12       Impact factor: 13.501

5.  Establishing clinical end points of respiratory function in large animals for clinical translation.

Authors:  Melissa A Goddard; Erin L Mitchell; Barbara K Smith; Martin K Childers
Journal:  Phys Med Rehabil Clin N Am       Date:  2011-12-15       Impact factor: 1.784

6.  Contraction force generated by tarsal joint flexion and extension in dogs with golden retriever muscular dystrophy.

Authors:  J N Kornegay; D J Bogan; J R Bogan; M K Childers; D D Cundiff; G F Petroski; R O Schueler
Journal:  J Neurol Sci       Date:  1999-07-01       Impact factor: 3.181

7.  A gene mutated in X-linked myotubular myopathy defines a new putative tyrosine phosphatase family conserved in yeast.

Authors:  J Laporte; L J Hu; C Kretz; J L Mandel; P Kioschis; J F Coy; S M Klauck; A Poustka; N Dahl
Journal:  Nat Genet       Date:  1996-06       Impact factor: 38.330

8.  Altered excitation-contraction coupling with skeletal muscle specific FKBP12 deficiency.

Authors:  Wei Tang; Christopher P Ingalls; William J Durham; Jessica Snider; Michael B Reid; Gangyi Wu; Martin M Matzuk; Susan L Hamilton
Journal:  FASEB J       Date:  2004-08-02       Impact factor: 5.191

Review 9.  Centronuclear (myotubular) myopathy.

Authors:  Heinz Jungbluth; Carina Wallgren-Pettersson; Jocelyn Laporte
Journal:  Orphanet J Rare Dis       Date:  2008-09-25       Impact factor: 4.123

10.  Loss of myotubularin function results in T-tubule disorganization in zebrafish and human myotubular myopathy.

Authors:  James J Dowling; Andrew P Vreede; Sean E Low; Elizabeth M Gibbs; John Y Kuwada; Carsten G Bonnemann; Eva L Feldman
Journal:  PLoS Genet       Date:  2009-02-06       Impact factor: 5.917

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  15 in total

1.  Gene therapy prolongs survival and restores function in murine and canine models of myotubular myopathy.

Authors:  Martin K Childers; Romain Joubert; Karine Poulard; Christelle Moal; Robert W Grange; Jonathan A Doering; Michael W Lawlor; Branden E Rider; Thibaud Jamet; Nathalie Danièle; Samia Martin; Christel Rivière; Thomas Soker; Caroline Hammer; Laetitia Van Wittenberghe; Mandy Lockard; Xuan Guan; Melissa Goddard; Erin Mitchell; Jane Barber; J Koudy Williams; David L Mack; Mark E Furth; Alban Vignaud; Carole Masurier; Fulvio Mavilio; Philippe Moullier; Alan H Beggs; Anna Buj-Bello
Journal:  Sci Transl Med       Date:  2014-01-22       Impact factor: 17.956

Review 2.  Gene therapy for inherited muscle diseases: where genetics meets rehabilitation medicine.

Authors:  Robynne Braun; Zejing Wang; David L Mack; Martin K Childers
Journal:  Am J Phys Med Rehabil       Date:  2014-11       Impact factor: 2.159

3.  Ultrasound assessment of the diaphragm: Preliminary study of a canine model of X-linked myotubular myopathy.

Authors:  Aarti Sarwal; Michael S Cartwright; Francis O Walker; Erin Mitchell; Anna Buj-Bello; Alan H Beggs; Martin K Childers
Journal:  Muscle Nerve       Date:  2014-08-30       Impact factor: 3.217

4.  Long-term effects of systemic gene therapy in a canine model of myotubular myopathy.

Authors:  Matthew Elverman; Melissa A Goddard; David Mack; Jessica M Snyder; Michael W Lawlor; Hui Meng; Alan H Beggs; Ana Buj-Bello; Karine Poulard; Anthony P Marsh; Robert W Grange; Valerie E Kelly; Martin K Childers
Journal:  Muscle Nerve       Date:  2017-05-22       Impact factor: 3.217

5.  Guiding intramuscular diaphragm injections using real-time ultrasound and electromyography.

Authors:  Aarti Sarwal; Michael S Cartwright; Erin Mitchell; Koudy Williams; Francis O Walker; Martin K Childers
Journal:  Muscle Nerve       Date:  2015-02       Impact factor: 3.217

6.  Validity of a Neurological Scoring System for Canine X-Linked Myotubular Myopathy.

Authors:  Jessica M Snyder; Allison Meisner; David Mack; Melissa Goddard; Ian T Coulter; Robert Grange; Martin K Childers
Journal:  Hum Gene Ther Clin Dev       Date:  2015-06       Impact factor: 5.032

7.  Gait characteristics in a canine model of X-linked myotubular myopathy.

Authors:  Melissa A Goddard; Emily Burlingame; Alan H Beggs; Anna Buj-Bello; Martin K Childers; Anthony P Marsh; Valerie E Kelly
Journal:  J Neurol Sci       Date:  2014-08-29       Impact factor: 3.181

8.  Muscle pathology, limb strength, walking gait, respiratory function and neurological impairment establish disease progression in the p.N155K canine model of X-linked myotubular myopathy.

Authors:  Melissa A Goddard; David L Mack; Stefan M Czerniecki; Valerie E Kelly; Jessica M Snyder; Robert W Grange; Michael W Lawlor; Barbara K Smith; Alan H Beggs; Martin K Childers
Journal:  Ann Transl Med       Date:  2015-10

Review 9.  Gene therapy in monogenic congenital myopathies.

Authors:  Xuan Guan; Melissa A Goddard; David L Mack; Martin K Childers
Journal:  Methods       Date:  2015-10-14       Impact factor: 3.608

Review 10.  Adeno-associated virus-mediated gene therapy for metabolic myopathy.

Authors:  Cathryn S Mah; Meghan S Soustek; A Gary Todd; Angela McCall; Barbara K Smith; Manuela Corti; Darin J Falk; Barry J Byrne
Journal:  Hum Gene Ther       Date:  2013-11       Impact factor: 5.695

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