Literature DB >> 34297919

Genetic diseases in the omics era.

Jean-Baptiste Dupont1.   

Abstract

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Year:  2021        PMID: 34297919      PMCID: PMC8353239          DOI: 10.1016/j.ymthe.2021.07.007

Source DB:  PubMed          Journal:  Mol Ther        ISSN: 1525-0016            Impact factor:   12.910


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  7 in total

1.  Gene expression analyses in X-linked myotubular myopathy.

Authors:  S Noguchi; M Fujita; K Murayama; R Kurokawa; I Nishino
Journal:  Neurology       Date:  2005-09-13       Impact factor: 9.910

2.  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

3.  Systemic AAV8-Mediated Gene Therapy Drives Whole-Body Correction of Myotubular Myopathy in Dogs.

Authors:  David L Mack; Karine Poulard; Melissa A Goddard; Virginie Latournerie; Jessica M Snyder; Robert W Grange; Matthew R Elverman; Jérôme Denard; Philippe Veron; Laurine Buscara; Christine Le Bec; Jean-Yves Hogrel; Annie G Brezovec; Hui Meng; Lin Yang; Fujun Liu; Michael O'Callaghan; Nikhil Gopal; Valerie E Kelly; Barbara K Smith; Jennifer L Strande; Fulvio Mavilio; Alan H Beggs; Federico Mingozzi; Michael W Lawlor; Ana Buj-Bello; Martin K Childers
Journal:  Mol Ther       Date:  2017-02-22       Impact factor: 11.454

4.  Multi-omics comparisons of different forms of centronuclear myopathies and the effects of several therapeutic strategies.

Authors:  Sarah Djeddi; David Reiss; Alexia Menuet; Sébastien Freismuth; Juliana de Carvalho Neves; Sarah Djerroud; Xènia Massana-Muñoz; Anne-Sophie Sosson; Christine Kretz; Wolfgang Raffelsberger; Céline Keime; Olivier M Dorchies; Julie Thompson; Jocelyn Laporte
Journal:  Mol Ther       Date:  2021-05-01       Impact factor: 12.910

5.  AAV-Mediated Gene Transfer Restores a Normal Muscle Transcriptome in a Canine Model of X-Linked Myotubular Myopathy.

Authors:  Jean-Baptiste Dupont; Jianjun Guo; Edith Renaud-Gabardos; Karine Poulard; Virginie Latournerie; Michael W Lawlor; Robert W Grange; John T Gray; Ana Buj-Bello; Martin K Childers; David L Mack
Journal:  Mol Ther       Date:  2019-11-11       Impact factor: 11.454

Review 6.  Congenital myopathies: disorders of excitation-contraction coupling and muscle contraction.

Authors:  Heinz Jungbluth; Susan Treves; Francesco Zorzato; Anna Sarkozy; Julien Ochala; Caroline Sewry; Rahul Phadke; Mathias Gautel; Francesco Muntoni
Journal:  Nat Rev Neurol       Date:  2018-02-02       Impact factor: 42.937

Review 7.  Centronuclear myopathies under attack: A plethora of therapeutic targets.

Authors:  Hichem Tasfaout; Belinda S Cowling; Jocelyn Laporte
Journal:  J Neuromuscul Dis       Date:  2018
  7 in total

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