V. Wally, . Murauer, and J. Bauer, Spliceosome-Mediated Trans-Splicing: The Therapeutic Cut and Paste, Journal of Investigative Dermatology, vol.132, issue.8, pp.1959-1966, 2012.
DOI : 10.1038/jid.2012.101

URL : http://doi.org/10.1038/jid.2012.101

L. Mitchell, . Mcgarrity, and . Gj, Gene Therapy Progress and Prospects: Reprograming gene expression by trans-splicing, Gene Therapy, vol.126, issue.99, pp.1477-1485, 2005.
DOI : 10.5483/BMBRep.2003.36.6.538

URL : http://www.nature.com/gt/journal/v12/n20/pdf/3302596a.pdf

L. Roy, F. Charton, K. Lorson, and I. Richard, RNA-targeting approaches for neuromuscular diseases, Trends in Molecular Medicine, vol.15, issue.12, pp.580-591, 2009.
DOI : 10.1016/j.molmed.2009.10.005

S. Mansfield, . Chao, and C. Walsh, RNA repair using spliceosome-mediated RNA trans-splicing, Trends in Molecular Medicine, vol.10, issue.6, pp.263-268, 2004.
DOI : 10.1016/j.molmed.2004.04.007

H. Chao, . Mansfield, . Sg, R. Bartel, S. Hiriyanna et al., Phenotype correction of hemophilia A mice by spliceosome-mediated RNA trans-splicing, Nature Medicine, vol.9, issue.8, pp.1015-1019, 2003.
DOI : 10.1038/nm900

X. Liu, Q. Jiang, . Mansfield, . Sg, M. Puttaraju et al., Partial correction of endogenous DeltaF508 CFTR in human cystic fibrosis airway epithelia by spliceosome-mediated RNA trans-splicing, Nat Biotechnol, vol.20, pp.47-52, 2002.

T. Coady, . Baughan, . Td, M. Shababi, . Passini et al., Development of a Single Vector System that Enhances Trans-Splicing of SMN2 Transcripts, PLoS ONE, vol.102, issue.10, p.3468, 2008.
DOI : 10.1371/journal.pone.0003468.s002

T. Coady and C. Lorson, Trans-Splicing-Mediated Improvement in a Severe Mouse Model of Spinal Muscular Atrophy, Journal of Neuroscience, vol.30, issue.1, pp.126-130, 2010.
DOI : 10.1523/JNEUROSCI.4489-09.2010

T. Rodriguez-martin, K. Anthony, M. Garcia-blanco, . Mansfield, . Sg et al., Correction of tau mis-splicing caused by FTDP-17 MAPT mutations by spliceosome-mediated RNA trans-splicing, Human Molecular Genetics, vol.18, issue.17, pp.3266-3273, 2009.
DOI : 10.1093/hmg/ddp264

M. Avale, . Rodríguez-martín, and J. Gallo, Trans-splicing correction of tau isoform imbalance in a mouse model of tau mis-splicing, Human Molecular Genetics, vol.22, issue.13, pp.2603-2611, 2013.
DOI : 10.1093/hmg/ddt108

V. Wally, A. Klausegger, U. Koller, H. Lochmüller, S. Krause et al., 5??? Trans-Splicing Repair of the PLEC1 Gene, Journal of Investigative Dermatology, vol.128, issue.3, pp.568-574, 2008.
DOI : 10.1038/sj.jid.5701152

H. Rindt, . Yen, . Pf, . Thebeau, . Cn et al., Replacement of huntingtin exon 1 by trans-splicing, Cellular and Molecular Life Sciences, vol.128, issue.24, pp.4191-4204, 2012.
DOI : 10.1038/sj.jid.5701152

B. Gersh, . Maron, . Bj, . Bonow, . Ro et al., American Heart Association Task Force on Practice Guidelines 2011 ACCF/AHA Guideline for the Diagnosis and Treatment of Hypertrophic Cardiomyopathy: a report of the American College of Cardiology Foundation/American Heart Association Task Force on Practice Guidelines. Developed in collaboration with the American Association for Thoracic Surgery, Society for Cardiovascular Angiography and Interventions, and Society of Thoracic Surgeons, pp.212-260, 2011.

P. Elliott, B. Andersson, E. Arbustini, Z. Bilinska, F. Cecchi et al., Classification of the cardiomyopathies: a position statement from the european society of cardiology working group on myocardial and pericardial diseases, European Heart Journal, vol.29, issue.2, pp.270-276, 2008.
DOI : 10.1093/eurheartj/ehm342

P. Ehlermann, D. Weichenhan, J. Zehelein, H. Steen, R. Pribe et al., Adverse events in families with hypertrophic or dilated cardiomyopathy and mutations in the MYBPC3gene, BMC Medical Genetics, vol.100, issue.1, p.95, 2008.
DOI : 10.1172/JCI119555

S. Schlossarek, . Mearini, and L. Carrier, Cardiac myosin-binding protein C in hypertrophic cardiomyopathy: Mechanisms and therapeutic opportunities, Journal of Molecular and Cellular Cardiology, vol.50, issue.4, pp.613-620, 2011.
DOI : 10.1016/j.yjmcc.2011.01.014

P. Richard, P. Charron, L. Carrier, C. Ledeuil, T. Cheav et al., Hypertrophic cardiomyopathy: distribution of disease genes, spectrum of mutations, and implications for a molecular diagnosis strategy, EUROGENE Heart Failure Project. Circulation, vol.107, pp.2227-2232, 2003.

O. Cazorla, S. Szilagyi, N. Vignier, G. Salazar, E. Krämer et al., Length and protein kinase A modulations of myocytes in cardiac myosin binding protein C-deficient mice, Cardiovascular Research, vol.69, issue.2, pp.370-380, 2006.
DOI : 10.1016/j.cardiores.2005.11.009

N. Vignier, S. Schlossarek, B. Fraysse, G. Mearini, E. Krämer et al., Nonsense-Mediated mRNA Decay and Ubiquitin-Proteasome System Regulate Cardiac Myosin-Binding Protein C Mutant Levels in Cardiomyopathic Mice, Circulation Research, vol.105, issue.3, pp.239-248, 2009.
DOI : 10.1161/CIRCRESAHA.109.201251

I. Olivotto, F. Girolami, . Ackerman, . Mj, S. Nistri et al., Myofilament Protein Gene Mutation Screening and Outcome of Patients With Hypertrophic Cardiomyopathy, Mayo Clinic Proceedings, vol.83, issue.6, pp.630-638, 2008.
DOI : 10.1016/S0025-6196(11)60890-2

B. Fraysse, F. Weinberger, . Bardswell, . Sc, F. Cuello et al., Increased myofilament Ca2+ sensitivity and diastolic dysfunction as early consequences of Mybpc3 mutation in heterozygous knock-in mice, Journal of Molecular and Cellular Cardiology, vol.52, issue.6, pp.1299-1307, 2012.
DOI : 10.1016/j.yjmcc.2012.03.009

R. Brinster, J. Allen, R. Behringer, . Gelinas, and R. Palmiter, Introns increase transcriptional efficiency in transgenic mice., Proceedings of the National Academy of Sciences, vol.85, issue.3, pp.836-840, 1988.
DOI : 10.1073/pnas.85.3.836

URL : https://www.ncbi.nlm.nih.gov/pmc/articles/PMC279650/pdf

R. Palmiter, . Sandgren, . Ep, . Avarbock, A. Mr et al., Heterologous introns can enhance expression of transgenes in mice., Proceedings of the National Academy of Sciences, vol.88, issue.2, pp.478-482, 1991.
DOI : 10.1073/pnas.88.2.478

S. Lorain, C. Peccate, L. Hir, and L. Garcia, Exon Exchange Approach to Repair Duchenne Dystrophin Transcripts, PLoS ONE, vol.8, issue.5, p.10894, 2010.
DOI : 10.1371/journal.pone.0010894.s004

URL : http://doi.org/10.1371/journal.pone.0010894

H. Fuke and M. Ohno, Role of poly (A) tail as an identity element for mRNA nuclear export, Nucleic Acids Research, vol.36, issue.3, pp.1037-1049, 2008.
DOI : 10.1093/nar/gkm1120

O. Müller, B. Leuchs, . Pleger, . St, D. Grimm et al., Improved cardiac gene transfer by transcriptional and transductional targeting of adeno-associated viral vectors, Cardiovascular Research, vol.70, issue.1, pp.70-78, 2006.
DOI : 10.1016/j.cardiores.2005.12.017

F. Friedrich, . Wilding, . Br, S. Reischmann, C. Crocini et al., Evidence for FHL1 as a novel disease gene for isolated hypertrophic cardiomyopathy, Human Molecular Genetics, vol.21, issue.14, pp.3237-3254, 2012.
DOI : 10.1093/hmg/dds157

URL : https://academic.oup.com/hmg/article-pdf/21/14/3237/17256603/dds157.pdf

H. Chen, P. Kathirvel, Y. Lai, and P. , Correction of dystrophia myotonica type 1 pre-mRNA transcripts by artificial trans-splicing, Gene Therapy, vol.24, issue.2, pp.211-217, 2009.
DOI : 10.1038/6986

Y. Taniguchi, . Choi, . Pj, . Li, . Gw et al., Quantifying E. coli Proteome and Transcriptome with Single-Molecule Sensitivity in Single Cells, Science, vol.217, issue.2, pp.533-538, 2010.
DOI : 10.1126/science.280.5363.585

Y. Zhang and R. Friedlander, Using non-coding small RNAs to develop therapies for Huntington's disease, Gene Therapy, vol.309, issue.12, pp.1139-1149, 2011.
DOI : 10.1007/s00439-011-0995-8

F. Friedrich and L. Carrier, Genetics of Hypertrophic and Dilated Cardiomyopathy, Current Pharmaceutical Biotechnology, vol.13, issue.13, pp.2467-2476, 2012.
DOI : 10.2174/1389201011208062467

D. Brito, G. Miltenberger-miltenyi, V. Pereira, S. Silva, D. Diogo et al., Sarcomeric hypertrophic cardiomyopathy: genetic profile in a Portuguese population, Rev Port Cardiol, vol.31, pp.577-587, 2012.

K. Curila, L. Benesova, M. Penicka, M. Minarik, D. Zemanek et al., Spectrum and clinical manifestations of mutations in genes responsible for hypertrophic cardiomyopathy, Acta Cardiologica, vol.67, issue.1, pp.23-29, 2012.
DOI : 10.1080/AC.67.1.2146562

D. Grimm, . Kay, . Ma, and J. Kleinschmidt, Helper virus-free, optically controllable, and two-plasmid-based production of adeno-associated virus vectors of serotypes 1 to 6, Molecular Therapy, vol.7, issue.6, pp.839-850, 2003.
DOI : 10.1016/S1525-0016(03)00095-9

J. Grieger, . Choi, . Vw, and R. Samulski, Production and characterization of adeno-associated viral vectors, Nature Protocols, vol.221, issue.3, pp.1412-1428, 2006.
DOI : 10.1038/nprot.2006.207

M. Sands and J. Barker, Percutaneous intravenous injection in neonatal mice, Lab Anim Sci, vol.49, pp.328-330, 1999.