R. Bashir, S. Britton, T. Strachan, S. Keers, E. Vafiadaki et al., A gene related to Caenorhabditis elegans spermatogenesis factor fer-1 is mutated in limb-girdle muscular dystrophy type 2B, Nat Genet, vol.2010, issue.1, pp.37-42, 1998.

J. Liu, M. Aoki, I. Illa, C. Wu, M. Fardeau et al., Dysferlin, a novel skeletal muscle gene, is mutated in Miyoshi myopathy and limb girdle muscular dystrophy, Nature Genetics, vol.43, issue.1, pp.31-37, 1998.
DOI : 10.1002/ana.410430514

K. Nguyen, G. Bassez, M. Krahn, R. Bernard, P. Laforet et al., Phenotypic Study in 40 Patients With Dysferlin Gene Mutations, Archives of Neurology, vol.64, issue.8, pp.1176-82, 2007.
DOI : 10.1001/archneur.64.8.1176
URL : https://jamanetwork.com/journals/jamaneurology/articlepdf/794290/noc60143_1176_1182.pdf

D. Bansal, K. Miyake, S. Vogel, S. Groh, C. Chen et al., Defective membrane repair in dysferlin-deficient muscular dystrophy, Nature, vol.137, issue.6936, pp.168-72, 2003.
DOI : 10.1083/jcb.137.3.685

A. Defour, J. Van-der-meulen, R. Bhat, A. Bigot, R. Bashir et al., Dysferlin regulates cell membrane repair by facilitating injury-triggered acid sphingomyelinase secretion, Cell Death and Disease, vol.497, issue.6, p.1306, 2014.
DOI : 10.1113/jphysiol.1996.sp021790
URL : https://hal.archives-ouvertes.fr/hal-01311583

M. Krahn, N. Wein, M. Bartoli, W. Lostal, S. Courrier et al., A Naturally Occurring Human Minidysferlin Protein Repairs Sarcolemmal Lesions in a Mouse Model of Dysferlinopathy, Science Translational Medicine, vol.106, issue.1, pp.50-69, 2010.
DOI : 10.1016/S0166-0934(02)00138-6
URL : https://hal.archives-ouvertes.fr/hal-01610037

A. Lek, F. Evesson, F. Lemckert, G. Redpath, A. Lueders et al., Calpains, cleaved mini-dysferlinC72, and Ltype channels underpin calcium-dependent muscle membrane repair, J Neurosci, vol.3322, issue.12, pp.5085-94, 2013.
DOI : 10.1523/jneurosci.3560-12.2013
URL : http://www.jneurosci.org/content/jneuro/33/12/5085.full.pdf

A. Marg, V. Schoewel, T. Timmel, A. Schulze, C. Shah et al., Sarcolemmal Repair Is a Slow Process and Includes EHD2, Traffic, vol.64, issue.9, 2012.
DOI : 10.1063/1.1143970
URL : http://onlinelibrary.wiley.com/doi/10.1111/j.1600-0854.2012.01386.x/pdf

J. Mcdade, A. Archambeau, and D. Michele, Rapid actin-cytoskeleton-dependent recruitment of plasma membrane-derived dysferlin at wounds is critical for muscle membrane repair, The FASEB Journal, vol.28, issue.8, pp.3660-70, 2014.
DOI : 10.1096/fj.14-250191

A. Posey, . Jr, P. Pytel, K. Gardikiotes, A. Demonbreun et al., Endocytic Recycling Proteins EHD1 and EHD2 Interact with Fer-1-like-5 (Fer1L5) and Mediate Myoblast Fusion, Journal of Biological Chemistry, vol.11, issue.9, pp.7379-88, 2010.
DOI : 10.1016/j.bbrc.2004.12.182
URL : http://www.jbc.org/content/286/9/7379.full.pdf

G. Redpath, N. Woolger, A. Piper, F. Lemckert, A. Lek et al., Calpain cleavage within dysferlin exon 40a releases a synaptotagmin-like module for membrane repair, Molecular Biology of the Cell, vol.11, issue.10, pp.3037-3085, 2014.
DOI : 10.1242/jcs.02980
URL : http://www.molbiolcell.org/content/25/19/3037.full.pdf

J. Roche, R. Lovering, R. Roche, L. Ru, P. Reed et al., Extensive mononuclear infiltration and myogenesis characterize recovery of dysferlin-null skeletal muscle from contraction-induced injuries, AJP: Cell Physiology, vol.298, issue.2, pp.298-312, 2009.
DOI : 10.1152/ajpcell.00122.2009

N. De-luna, E. Gallardo, M. Soriano, R. Dominguez-perles, C. De-la-torre et al., Absence of dysferlin alters myogenin expression and delays human muscle differentiation " in vitro Impaired muscle growth and response to insulin-like growth factor 1 in dysferlin-mediated muscular dystrophy, J Biol Chem. Hum Mol Genet, vol.2812012, issue.254, pp.17092-8779, 2006.

A. Balasubramanian, G. Kawahara, V. Gupta, A. Rozkalne, A. Beauvais et al., Fam65b is important for formation of the HDAC6-dysferlin protein complex during myogenic cell differentiation, The FASEB Journal, vol.28, issue.7, pp.2955-6902, 2014.
DOI : 10.1096/fj.13-246470

W. Grose, K. Clark, D. Griffin, V. Malik, K. Shontz et al., Homologous Recombination Mediates Functional Recovery of Dysferlin Deficiency following AAV5 Gene Transfer, PLoS ONE, vol.179, issue.6, p.39233, 2012.
DOI : 10.1371/journal.pone.0039233.s002

W. Lostal, M. Bartoli, N. Bourg, C. Roudaut, A. Bentaib et al., Efficient recovery of dysferlin deficiency by dual adeno-associated vector-mediated gene transfer, Hum Mol Genet, vol.1916, issue.10, pp.1897-907, 2010.
URL : https://hal.archives-ouvertes.fr/hal-01610036

S. Philippi, S. Lorain, C. Beley, C. Peccate, G. Precigout et al., Dysferlin rescue by spliceosome-mediated pre-mRNA trans-splicing targeting introns harbouring weakly defined 3??? splice sites, Human Molecular Genetics, vol.24, issue.14, 2015.
DOI : 10.1093/hmg/ddv141
URL : https://academic.oup.com/hmg/article-pdf/24/14/4049/17262161/ddv141.pdf

P. Sondergaard, D. Griffin, E. Pozsgai, R. Johnson, W. Grose et al., AAV.Dysferlin Overlap Vectors Restore Function in Dysferlinopathy Animal Models, Annals of Clinical and Translational Neurology, vol.68, issue.3, pp.256-7031, 2015.
DOI : 10.1002/ana.22251

S. Cirak, V. Arechavala-gomeza, M. Guglieri, L. Feng, S. Torelli et al., Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study, The Lancet, vol.378, issue.9791, pp.595-60507, 2011.
DOI : 10.1016/S0140-6736(11)60756-3

J. Van-deutekom, A. Janson, I. Ginjaar, W. Frankhuizen, A. Aartsma-rus et al., Local Dystrophin Restoration with Antisense Oligonucleotide PRO051, New England Journal of Medicine, vol.357, issue.26, pp.2677-86, 2007.
DOI : 10.1056/NEJMoa073108

T. Voit, H. Topaloglu, V. Straub, F. Muntoni, N. Deconinck et al., Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study, The Lancet Neurology, vol.13, issue.10, pp.987-96, 2014.
DOI : 10.1016/S1474-4422(14)70195-4

K. Flanigan, T. Voit, X. Rosales, L. Servais, J. Kraus et al., Pharmacokinetics and safety of single doses of drisapersen in non-ambulant subjects with Duchenne muscular dystrophy: Results of a double-blind randomized clinical trial, Neuromuscular Disorders, vol.24, issue.1, pp.16-24, 2013.
DOI : 10.1016/j.nmd.2013.09.004

M. Sinnreich, C. Therrien, and G. Karpati, Lariat branch point mutation in the dysferlin gene with mild limbgirdle muscular dystrophy, Neurology, vol.6612, issue.7, pp.1114-1120, 2006.

N. Wein, A. Avril, M. Bartoli, C. Beley, S. Chaouch et al., Efficient bypass of mutations in dysferlin deficient patient cells by antisense-induced exon skipping, Hum Mutat, vol.3112, issue.2, pp.136-4203, 2009.
URL : https://hal.archives-ouvertes.fr/hal-01610031

L. Cartegni, J. Wang, Z. Zhu, M. Zhang, and A. Krainer, ESEfinder: a web resource to identify exonic splicing enhancers, Nucleic Acids Research, vol.31, issue.13, pp.3568-71, 2003.
DOI : 10.1093/nar/gkg616
URL : https://academic.oup.com/nar/article-pdf/31/13/3568/9487615/gkg616.pdf

F. Desmet and C. Beroud, Bioinformatics and Mutations Leading to Exon Skipping, Methods Mol Biol, vol.867, pp.17-3529, 2012.
DOI : 10.1007/978-1-61779-767-5_2

B. Sinha, D. Koster, R. Ruez, P. Gonnord, M. Bastiani et al., Cells Respond to Mechanical Stress by Rapid Disassembly of Caveolae, Cell, vol.144, issue.3, pp.402-415, 2011.
DOI : 10.1016/j.cell.2010.12.031
URL : https://hal.archives-ouvertes.fr/hal-00821331

A. Aartsma-rus, W. Kaman, M. Bremmer-bout, A. Janson, J. Den-dunnen et al., Comparative analysis of antisense oligonucleotide analogs for targeted DMD exon 46 skipping in muscle cells, Gene Therapy, vol.40, issue.18, pp.1391-80702, 2004.
DOI : 10.1021/bi011279e

A. Aartsma-rus, Overview on DMD Exon Skipping, Methods Mol Biol, vol.867, pp.97-11629, 2012.
DOI : 10.1007/978-1-61779-767-5_7

B. Wang, Z. Yang, B. Brisson, H. Feng, Z. Zhang et al., Membrane blebbing as an assessment of functional rescue of dysferlin-deficient human myotubes via nonsense suppression, Journal of Applied Physiology, vol.109, issue.3, pp.901-906, 2010.
DOI : 10.1152/japplphysiol.01366.2009

N. Oulhen, T. Onorato, I. Ramos, and G. Wessel, Dysferlin is essential for endocytosis in the sea star oocyte, Developmental Biology, vol.388, issue.1, pp.94-102, 2013.
DOI : 10.1016/j.ydbio.2013.12.018

J. Kerr, A. Ziman, A. Mueller, J. Muriel, E. Kleinhans-welte et al., Dysferlin stabilizes stress-induced Ca2+ signaling in the transverse tubule membrane, Proceedings of the National Academy of Sciences, vol.39, issue.2, pp.20831-605, 2013.
DOI : 10.1002/mus.21188

J. Dominov, O. Uyan, P. Sapp, D. Mckenna-yasek, B. Nallamilli et al., A novel dysferlin mutant pseudoexon bypassed with antisense oligonucleotides. Annals of clinical and translational neurology, pp.703-723, 2014.
DOI : 10.1002/acn3.96
URL : http://onlinelibrary.wiley.com/doi/10.1002/acn3.96/pdf

N. Goemans, M. Tulinius, J. Van-den-akker, B. Burm, P. Ekhart et al., Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy, New England Journal of Medicine, vol.364, issue.16, pp.1513-1535, 2011.
DOI : 10.1056/NEJMoa1011367

V. Arechavala-gomeza, K. Anthony, J. Morgan, and F. Muntoni, Antisense Oligonucleotide-Mediated Exon Skipping for Duchenne Muscular Dystrophy: Progress and Challenges, Current Gene Therapy, vol.12, issue.3, pp.152-60, 2012.
DOI : 10.2174/156652312800840621

B. Azakir, D. Fulvio, S. Salomon, S. Brockhoff, M. Therrien et al., Modular dispensability of dysferlin's C2 domains reveals rational design for mini-dysferlin molecules, J Biol Chem, vol.28, issue.06, 2012.

A. Aartsma-rus, K. Singh, I. Fokkema, I. Ginjaar, G. Van-ommen et al., Therapeutic exon skipping for dysferlinopathies?, Eur J Hum Genet, vol.1811, issue.8, pp.889-94, 2010.
DOI : 10.1038/ejhg.2010.4
URL : https://hal.archives-ouvertes.fr/hal-00509049

A. Aartsma-rus, A. Janson, W. Kaman, M. Bremmer-bout, G. Van-ommen et al., Antisense-Induced Multiexon Skipping for Duchenne Muscular Dystrophy Makes More Sense, The American Journal of Human Genetics, vol.74, issue.1, pp.83-92, 2003.
DOI : 10.1086/381039
URL : https://doi.org/10.1086/381039

A. Goyenvalle, J. Wright, A. Babbs, V. Wilkins, L. Garcia et al., Engineering Multiple U7snRNA Constructs to Induce Single and Multiexon-skipping for Duchenne Muscular Dystrophy, Molecular Therapy, vol.20, issue.6, pp.1212-1233, 2012.
DOI : 10.1038/mt.2012.26
URL : https://doi.org/10.1038/mt.2012.26

Y. Echigoya and T. Yokota, Skipping Multiple Exons of Dystrophin Transcripts Using Cocktail Antisense Oligonucleotides, Nucleic Acid Therapeutics, vol.24, issue.1, pp.57-68, 2014.
DOI : 10.1089/nat.2013.0451

C. Adkin, P. Meloni, S. Fletcher, A. Adams, F. Muntoni et al., Multiple exon skipping strategies to bypass dystrophin mutations, Neuromuscul Disord, vol.221221, issue.4, pp.297-305, 2011.
DOI : 10.1016/j.nmd.2011.10.007
URL : https://doi.org/10.1016/j.nmd.2011.10.007

C. Beroud, S. Tuffery-giraud, M. Matsuo, D. Hamroun, V. Humbertclaude et al., Multiexon skipping leading to an artificial DMD protein lacking amino acids from exons 45 through 55 could rescue up to 63% of patients with Duchenne muscular dystrophy, Human Mutation, vol.18, issue.Web Server issu, pp.196-20217, 2006.
DOI : 10.1016/S0002-9440(10)65354-0
URL : https://hal.archives-ouvertes.fr/hal-00122078

A. Aartsma-rus, W. Kaman, R. Weij, J. Den-dunnen, G. Van-ommen et al., Exploring the Frontiers of Therapeutic Exon Skipping for Duchenne Muscular Dystrophy by Double Targeting within One or Multiple Exons, Molecular Therapy, vol.14, issue.3, pp.401-707, 2006.
DOI : 10.1016/j.ymthe.2006.02.022

F. Gualandi, C. Trabanelli, P. Rimessi, E. Calzolari, L. Toffolatti et al., Multiple exon skipping and RNA circularisation contribute to the severe phenotypic expression of exon 5 dystrophin deletion, Journal of Medical Genetics, vol.40, issue.8, p.100, 2003.
DOI : 10.1136/jmg.40.8.e100

V. Kergourlay, G. Rai, G. Blandin, D. Salgado, C. Beroud et al., Identification of Splicing Defects Caused by Mutations in the Dysferlin Gene, Human Mutation, vol.27, issue.12, pp.1532-4115, 2014.
DOI : 10.1002/humu.9424
URL : https://hal.archives-ouvertes.fr/hal-01610021