Research Topics
Genomes and Genes | Jocelyn LaporteSummaryCountry: France Publications
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Publications
AAV-mediated intramuscular delivery of myotubularin corrects the myotubular myopathy phenotype in targeted murine muscle and suggests a function in plasma membrane homeostasisAnna Buj-Bello
Department of Neurobiology and Genetics, INSERM U596, CNRS UMR 7104, Universite Louis Pasteur de Strasbourg, College de France, 67404 Illkirch, France
Hum Mol Genet 17:2132-43. 2008..This study provides a proof-of-principle that local delivery of an AAV vector expressing myotubularin can improve the motor capacities of XLMTM muscle and represents a novel approach to study myotubularin function in skeletal muscle...
Centronuclear (myotubular) myopathyHeinz Jungbluth
Department of Paediatric Neurology, Neuromuscular Service, Evelina Children s Hospital, St Thomas Hospital, London, UK
Orphanet J Rare Dis 3:26. 2008..Whereas the X-linked form due to MTM1 mutations is often fatal in infancy, dominant forms due to DNM2 mutations and some cases of the recessive BIN1-related form appear to be associated with an overall more favourable prognosis...
Phosphatase-dead myotubularin ameliorates X-linked centronuclear myopathy phenotypes in miceLeonela Amoasii
Department of Translational Medecine, Institut de Genetique et de Biologie Moleculaire et Cellulaire IGBMC, INSERM U964, CNRS UMR7104, Universite de Strasbourg, College de France, Illkirch, France
PLoS Genet 8:e1002965. 2012..This underlines that removal of enzymes should be used with care to conclude on the physiological importance of their activity...
Constitutive Activation of the Calcium Sensor STIM1 Causes Tubular-Aggregate MyopathyJohann Böhm
Département de Médecine Translationelle et Neurogénétique, Institut de Genetique et de Biologie Moleculaire et Cellulaire, 67404 Illkirch, France Unité 964, Institut National de la Sante et de la Recherche Medicale, 67404 Illkirch, France Unité Mixte de Recherche 7104, Centre National de la Recherche Scientifique, 67404 Illkirch, France Université de Strasbourg, 67404 Illkirch, France Chaire de Génétique Humaine, College de France, 67404 Illkirch, France
Am J Hum Genet 92:271-8. 2013....
Myotubularin controls desmin intermediate filament architecture and mitochondrial dynamics in human and mouse skeletal muscleKarim Hnia
Department of Neurobiology and Genetics, Institut de Genetique et de Biologie Moleculaire et Cellulaire, Illkirch, France
J Clin Invest 121:70-85. 2011..Defects in IF stabilization and mitochondrial dynamics appear as common physiopathological features of centronuclear myopathies and desmin-related myopathies...
T-tubule disorganization and defective excitation-contraction coupling in muscle fibers lacking myotubularin lipid phosphataseLama Al-Qusairi
Department of Neurobiology and Genetics, and Imaging Center Electron Microscopy, Institut de Genetique et de Biologie Moleculaire et Cellulaire, Institut National de la Santé et de la Recherche Médicale U964, Centre National de la Recherche Scientifique, Unite Mixte de Recherche 7104, Universite Louis Pasteur, College de France, 67404 Illkirch, France
Proc Natl Acad Sci U S A 106:18763-8. 2009....
Mutation spectrum in the large GTPase dynamin 2, and genotype-phenotype correlation in autosomal dominant centronuclear myopathyJohann Böhm
Department of Translational Medicine and Neurogenetics, Institut de Genetique et de Biologie Moleculaire et Cellulaire, INSERM U964 CNRS UMR7104, University of Strasbourg, College de France, Illkirch, France
Hum Mutat 33:949-59. 2012..Defects in membrane trafficking due to DNM2 mutations potentially represent a common pathological mechanism in CNM and CMT...
Defects in amphiphysin 2 (BIN1) and triads in several forms of centronuclear myopathiesAnne Toussaint
Department of Neurobiology and Genetics, Institut de Genetique et de Biologie Moleculaire et Cellulaire, Illkirch, France
Acta Neuropathol 121:253-66. 2011..We propose that aberrant BIN1 localization and defects in triad structure are part of a common pathogenetic mechanism shared between the three forms of centronuclear myopathies...
Impacts of massively parallel sequencing for genetic diagnosis of neuromuscular disordersNasim Vasli
IGBMC Institut de Génétique et de Biologie Moléculaire et Cellulaire, 1, rue Laurent Fries, BP 10142, 67404 Illkirch, France
Acta Neuropathol 125:173-85. 2013..Massively parallel sequencing for mutation identification is expected to greatly improve diagnosis, genetic counseling and patient management...
Myotubularin phosphoinositide phosphatases: cellular functions and disease pathophysiologyKarim Hnia
Department of Translational Medicine and Neurogenetics, IGBMC Institut de Génétique et de Biologie Moléculaire et Cellulaire, Illkirch, France
Trends Mol Med 18:317-27. 2012..A better understanding of myotubularin pathophysiology will pave the way towards therapeutic strategies...
Novel molecular diagnostic approaches for X-linked centronuclear (myotubular) myopathy reveal intronic mutationsValérie Tosch
Department of Neurobiology and Genetics, Institut de Genetique et de Biologie Moleculaire et Cellulaire, Illkirch, France
Neuromuscul Disord 20:375-81. 2010..Patients with centronuclear myopathy and no molecular diagnosis should be investigated for MTM1 defects at the cDNA and protein level...
T-tubule biogenesis and triad formation in skeletal muscle and implication in human diseasesLama Al-Qusairi
Department of Translational Medecine and Neurogenetics, IGBMC Institut de Génétique et de Biologie Moléculaire et Cellulaire, 1 rue Laurent Fries, 67404 Illkirch, France
Skelet Muscle 1:26. 2011..The importance of these proteins in triad biogenesis and subsequently in muscle contraction is sustained by studies on animal models and by the direct implication of most of these proteins in human myopathies...
