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Jacques-P. Tremblay

Professeur titulaire

jacques-p.tremblay@fmed.ulaval.ca

Site personnel

Département de médecine moléculaire

Centre thématique de recherche en neurosciences

Centre de recherche du CHU de Québec - Université Laval

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Contribution à la recherche

Thématiques de recherche de la Faculté de médecine

Médecine régénératrice et médecine moléculaire
Neurosciences et santé mentale

Domaines de recherche

Thérapie génique et moléculaire
Biotechnologies médicales, n.c.a.

Projets de recherche en cours

Correction by Prime editing of point mutations responsible for Cystic fibrosis in Quebec province - Génome Québec - Cibler des mutations rares, orphelines de CFTR, McGill University, co-chercheur - 2025-04-01 au 2028-03-30
In vivo correction by CRISPR PRIME editing of mutations responsible for Duchenne Muscular Dystrophy - Vaincre Duchenne Canada, chercheur principal - 2023-12-01 au 2026-11-30
Correction of point mutations responsible for Dysferlinopathy using Prime Editing Renouvellement 2025-2026 - Jain Foundation Inc., chercheur principal - 2025-02-01 au 2026-01-31

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Financements des 2 dernières années

Removal of the GAA repeat with the CRISPR/Cas9 system in Friedreich patient cells and in the YG8sR mouse model - Instituts de recherche en santé du Canada - Subvention Projet, chercheur principal - 2019-10-01 au 2024-09-30
Correcting point mutations responsible for Dysferlinopathy using Prime Editing - Jain Foundation Inc., chercheur principal - 2023-08-01 au 2024-07-31
Développement de microdispositifs transdermiques peu invasifs pour l’administration d'acides nucléiques : vaccination et thérapie génique - Fonds de recherche du Québec - Santé - Réseaux thématiques de recherche, Université de Montréal, co-chercheur - 2023-04-01 au 2024-03-31
Development of an AAV library - Conseil national de recherches du Canada - Programmes et initiatives de R&D collaboratifs, chercheur principal - 2022-04-01 au 2024-03-31
Développement d'un traitement pour prévenir ou ralentir la progression de la maladie d'Alzheimer - Fondation Famille Lemaire, chercheur principal - 2023-11-08 au 2024-03-31
Correction with the Prime editing technology of point mutations responsible for Duchenne Muscular Dystrophy - Instituts de recherche en santé du Canada - Subvention Projet, chercheur principal - 2023-03-01 au 2024-02-29
PRIME editing correction of the T4709M mutation responsible for some cases of Ryanodine receptor type I-related myopathies - RYR-1 Foundation, chercheur principal - 2021-07-01 au 2023-06-30
Development of a rapid and simple test to detect the COVID-19 variants that can be used in remote areas and developing countries - Instituts de recherche en santé du Canada - Subvention de fonctionnement : Possibilité de financement sur les nouveaux besoins prioritaires en recherche sur la COVID-19, chercheur principal - 2021-06-01 au 2022-05-31

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Publications

Reading frame correction by targeted genome editing restores dystrophin expression in cells from Duchenne muscular dystrophy patients, , Molecular therapy : the journal of the American Society of Gene Therapy, 2013, 10.1038/mt.2013.111
Not an inside job: how can transplantation of relatively few exogenous satellite cells do what thousands of endogenous cells cannot?, , Molecular therapy : the journal of the American Society of Gene Therapy, 2011, 10.1038/mt.2010.276
Combined immunosuppression of mycophenolate mofetil and FK506 for myoblast transplantation in mdx mice, , Transplantation, 2001, 10.1097/00007890-200107150-00010
Intramuscular transplantation of human postnatal myoblasts generates functional donor-derived satellite cells, , Molecular therapy : the journal of the American Society of Gene Therapy, 2010, 10.1038/mt.2010.128
Early and massive death of myoblasts transplanted into skeletal muscle : responsible factors and potential solutions, , Current opinion in organ transplantation, 2007
The paracrine effect: pivotal mechanism in cell-based cardiac repair, , Journal of cardiovascular translational research, 2010, 10.1007/s12265-010-9198-2
Small-molecule inhibitors of proteasome increase CjCas9 protein stability, Jacques P. Tremblay, Cedric Happi Mbakam, Nathalie Majeau, Pouiré Yaméogo, PLOS ONE, 2023, 10.1371/journal.pone.0280353
Ex vivo modification of cells to induce a muscle-based expression, , Current gene therapy, 2006, 10.2174/156652306779010651
Successful myoblast transplantation in primates depends on appropriate cell delivery and induction of regeneration in the host muscle, , Experimental neurology, 1999, 10.1006/exnr.1998.6973
Treatment with anti-CD154 antibody and donor-specific transfusion prevents acute rejection of myoblast transplantation, , Transplantation, 2002, 10.1097/00007890-200202150-00021
Ischemic central necrosis in pockets of transplanted myoblasts in nonhuman primates: implications for cell-transplantation strategies, , Transplantation, 2007, 10.1097/01.tp.0000288322.94252.22
Efficient myoblast transplantation in mice immunosuppressed with monoclonal antibodies and CTLA4 Ig, , Transplantation proceedings, 1997, 10.1016/s0041-1345(97)00166-8
Efficacy and site-specificity of adenoviral vector integration mediated by the phage φC31 integrase, , Human gene therapy methods, 2012, 10.1089/hgtb.2012.122
Vascular endothelial growth factor reduced hypoxia-induced death of human myoblasts and improved their engraftment in mouse muscles, , Gene therapy, 2008, 10.1038/sj.gt.3303059
Successful histocompatible myoblast transplantation in dystrophin-deficient mdx mouse despite the production of antibodies against dystrophin, , The Journal of cell biology, 1995, 10.1083/jcb.131.4.975
Cell Therapy in Myology: Dynamics of Muscle Precursor Cell Death after Intramuscular Administration in Non-human Primates, , Molecular therapy. Methods & clinical development, 2017, 10.1016/j.omtm.2017.05.002
Successful Correction by Prime Editing of a Mutation in the RYR1 Gene Responsible for a Myopathy, Jacques P. Tremblay, Joël Rousseau, Kelly Godbout, Cells, 2023, 10.3390/cells13010031
Normal myoblast implantation in MDX mice prevents muscle damage by exercise, , Biochemical and biophysical research communications, 1998, 10.1006/bbrc.1998.9276
Muscle glucose-6-phosphate dehydrogenase deficiency: restoration of enzymatic activity in hybrid myotubes, , Muscle & nerve, 1993, 10.1002/mus.880160604
Gaba and benzodiazepines in invertebrate species, , 1988
In vivo migration of transplanted myoblasts requires matrix metalloproteinase activity, , Experimental cell research, 2000, 10.1006/excr.2000.4962
Intramuscular cell transplantation as a potential treatment of myopathies: clinical and preclinical relevant data, , Expert opinion on biological therapy, 2011, 10.1517/14712598.2011.548800
Integration of a Raman spectroscopy system to a robotic-assisted surgical system for real-time tissue characterization during radical prostatectomy procedures, , Journal of biomedical optics, 2019, 10.1117/1.JBO.24.2.025001
Localization of dystrophin in the Purkinje cells of normal mice, , Neuroscience letters, 1992, 10.1016/0304-3940(92)90309-u
Delivery of RNAs to Specific Organs by Lipid Nanoparticles for Gene Therapy, Jacques P. Tremblay, Kelly Godbout, Pharmaceutics, 2022, 10.3390/pharmaceutics14102129
Control of inflammatory damage by anti-LFA-1: increase success of myoblast transplantation, , Cell transplantation, 1997
Distribution of spontaneous release along the frog neuromuscular junction, , Neuroscience letters, 1984, 10.1016/0304-3940(84)90559-7
The advances and challenges of Gene Therapy for Duchenne Muscular Dystrophy, , Journal of Genetic Medicine and Gene Therapy, 2017, 10.29328/journal.jgmgt.1001003
Mosaic expression of dystrophin in the cerebellum of heterozygote dystrophic (mdx) mice, , Neuromuscular disorders : NMD, 1992, 10.1016/s0960-8966(06)80002-7
FK506 immunosuppression to control the immune reactions triggered by first-generation adenovirus-mediated gene transfer, , Human gene therapy, 1995, 10.1089/hum.1995.6.11-1391
Dopamine, serotonin and related compounds: presynaptic effects on synaptic depression, frequency facilitation, and post-tetanic potentiation at a synapse in Aplysia californica, , Brain research, 1976, 10.1016/0006-8993(76)90380-2
Universal Prime Editing Therapeutic Strategy for RyR1-Related Myopathies: A Protective Mutation Rescues Leaky RyR1 Channel, Jacques P. Tremblay, Andrew R. Marks, Emmanuel Planel, Yaoyao Lu, Geoffrey Canet, Gabriel Lamothe, Anetta E. Wronska, Joël Rousseau, Alexia Falle, Sina Ramezani, Steven R. Reiken, Mathieu Dugas, Kelly Godbout, International Journal of Molecular Sciences, 2025, 10.3390/ijms26072835
Section VI - Novel Genetic Technologies. 6.2 Gene Therapy, , 2009
CD56+ Muscle Derived Cells but Not Retinal NG2+ Perivascular Cells of Nonhuman Primates are Myogenic after Intramuscular Transplantation in Immunodeficient Mice, , Journal of stem cell research & therapy, 2017, 10.4172/2157-7633.1000377
Macrophages improve survival, proliferation and migration of engrafted myogenic precursor cells into MDX skeletal muscle, , PloS one, 2012, 10.1371/journal.pone.0046698
The protective mutation A673T in amyloid precursor protein gene decreases Aβ peptides production for 14 forms of Familial Alzheimer's Disease in SH-SY5Y cells, , PloS one, 2020, 10.1371/journal.pone.0237122
Electrotransfer of the full-length dog dystrophin into mouse and dystrophic dog muscles, , Human gene therapy, 2010, 10.1089/hum.2010.024
Dystrophin expression in muscles of duchenne muscular dystrophy patients after high-density injections of normal myogenic cells, , Journal of neuropathology and experimental neurology, 2006, 10.1097/01.jnen.0000218443.45782.81
Current status of pharmaceutical and genetic therapeutic approaches to treat DMD, , Molecular therapy : the journal of the American Society of Gene Therapy, 2011, 10.1038/mt.2011.59
Ganglioside GM3 levels are altered in a mouse model of HIBM: GM3 as a cellular marker of the disease, , PloS one, 2010, 10.1371/journal.pone.0010055
Dystrophin expression following the transplantation of normal muscle precursor cells protects mdx muscle from contraction-induced damage, , Cell transplantation, 2010, 10.3727/096368910X4863235
Advances & challenges of using CRISPR-Cas9 gene editing for treating Duchenne muscular dystrophy, , Cell & gene therapy insights, 2017, 10.18609/cgti.2017.003
Intramuscular transplantation of myogenic cells in primates: importance of needle size, cell number, and injection volume, , Cell transplantation, 2014, 10.3727/096368912X661337
A monoclonal antibody to conotoxin reveals the distribution of a subset of calcium channels in the rat cerebellar cortex, , Brain research. Molecular brain research, 1991, 10.1016/0169-328x(91)90004-h
Prime editing optimized RTT permits the correction of the c.8713C>T mutation in DMD gene, Jacques P. Tremblay, Vincent Mouly, Kamel Mamchaoui, Anne Bigot, Yaoyao Lu, Joel Rousseau, Cedric Happi Mbakam, Molecular Therapy - Nucleic Acids, 2022, 10.1016/j.omtn.2022.09.022
Utilization of an antibody specific for human dystrophin to follow myoblast transplantation in nude mice, , Cell transplantation, 1993, 10.1177/096368979300200204
Morphological changes in presynaptic terminals of the chick ciliary ganglion after stimulation in vivo. A stereological study showing a net loss of total membrane, , Experimental brain research, 1981, 10.1007/BF00238388
Electroporation as a method to induce myofiber regeneration and increase the engraftment of myogenic cells in skeletal muscles of primates, , Journal of neuropathology and experimental neurology, 2013, 10.1097/NEN.0b013e31829bac22
Role of non-major histocompatibility complex antigens in the rejection of transplanted myoblasts, , Transplantation, 1997, 10.1097/00007890-199703270-00016
Myoblast transplantation in whole muscle of nonhuman primates, , Journal of neuropathology and experimental neurology, 2000, 10.1093/jnen/59.3.197
Prevention of immune reactions triggered by first-generation adenoviral vectors by monoclonal antibodies and CTLA4Ig, , Human gene therapy, 1996, 10.1089/hum.1996.7.12-1455
Use of normal and genetically modified myoblasts for the treatment of myopathies, , 2000
Improved success of myoblast transplantation in mdx mice by blocking the myostatin signal, , Transplantation, 2005, 10.1097/01.tp.0000167379.27872.2b
Proximodistal gradients of the postjunctional folds at the frog neuromuscular junction: a scanning electron microscopic study, , Neuroscience, 1989, 10.1016/0306-4522(89)90271-6
Non-uniform distribution of miniature endplate potential amplitudes along the length of the frog neuromuscular junction, , Neuroscience letters, 1987, 10.1016/0304-3940(87)90147-9
Transplantation of normal and DMD myoblasts expressing the telomerase gene in SCID mice, , Biochemical and biophysical research communications, 2000, 10.1006/bbrc.2000.2735
Overexpression of follistatin in human myoblasts increases their proliferation and differentiation, and improves the graft success in SCID mice, , Cell transplantation, 2009, 10.3727/096368909X470865
Myoblast survival enhancement and transplantation success improvement by heat-shock treatment in mdx mice, , Transplantation, 2004, 10.1097/01.tp.0000121503.01535.f5
Successful myoblast allotransplantation in mdx mice using rapamycin, , Transplantation, 1995
The CRISPR system can correct or modify the expression of genes responsible for hereditary diseases, , Medecine sciences : M/S, 2015, 10.1051/medsci/20153111016
Myopathy in a rhesus monkey with biopsy findings similar to human sporadic inclusion body myositis, , Neuromuscular disorders : NMD, 2013, 10.1016/j.nmd.2012.10.020
Generation of human endometrial knockout cell lines with the CRISPR/Cas9 system confirms the prostaglandin F2α synthase activity of aldo-ketoreductase 1B1, , Molecular human reproduction, 2014, 10.1093/molehr/gau023
Synaptic depression at a synapse in Aplysia californica: analysis in terms of a material flow model of neurotransmitter, , Brain research, 1976, 10.1016/0006-8993(76)90379-6
Endosome disruption enhances the functional nuclear delivery of Tat-fusion proteins, , Biochemical and biophysical research communications, 2004, 10.1016/j.bbrc.2004.04.180
Cerebroside may be falsely identified as a soluble 'brain specific protein', , Journal of neurochemistry, 1974, 10.1111/j.1471-4159.1974.tb04360.x
Dystrophin-like immunoreactivity in monkey and human brain areas involved in learning and motor functions, , Neuroscience letters, 1992, 10.1016/0304-3940(92)90890-j
Is the MEPP due to the release of one vesicle or to the simultaneous release of several vesicles at one active zone?, , Brain research, 1983, 10.1016/0165-0173(83)90009-7
Induction of tolerance across fully mismatched barriers by a nonmyeloablative treatment excluding antibodies or irradiation use, , Cell transplantation, 2006, 10.3727/000000006783981521
Decidualization and maintenance of a functional prostaglandin system in human endometrial cell lines following transformation with SV40 large T antigen, , Molecular human reproduction, 2006, 10.1093/molehr/gal034
Very efficient myoblast allotransplantation in mice under FK506 immunosuppression, , Muscle & nerve, 1994, 10.1002/mus.880171210
Human Muscle Precursor Cells Form Human-Derived Myofibers in Skeletal Muscles of Nonhuman Primates: A Potential New Preclinical Setting to Test Myogenic Cells of Human Origin for Cell Therapy of Myopathies, , Journal of neuropathology and experimental neurology, 2020, 10.1093/jnen/nlaa110
Non-uniform release at the frog neuromuscular junction: evidence of morphological and physiological plasticity, , Brain research, 1987, 10.1016/0165-0173(87)90019-1
Immune reactions after myoblast transplantation in mouse muscles, , Transplantation proceedings, 1992
Prime Editing for Human Gene Therapy: Where Are We Now?, Jacques P. Tremblay, Kelly Godbout, Cells, 2023, 10.3390/cells12040536
Anti-inflammatory effect of transforming growth factor-beta1 in myoblast transplantation, , Transplantation, 1998, 10.1097/00007890-199803270-00005
Partial laminin alpha2 chain restoration in alpha2 chain-deficient dy/dy mouse by primary muscle cell culture transplantation, , The Journal of cell biology, 1996, 10.1083/jcb.133.1.185
A Comment on Muscle Xenografts Reproduce Key Molecular Features of Facioscapulohumeral Muscular Dystrophy : What Is New and What Has Already been Done and Reported but Was Not Quoted?, , Cell transplantation, 2020, 10.1177/0963689720939120
Successful transplantation of genetically corrected DMD myoblasts following ex vivo transduction with the dystrophin minigene, , Biochemical and biophysical research communications, 1998, 10.1006/bbrc.1998.8739
Cytoplasmic restoration and persistence of glucose-6-phosphate dehydrogenase activity in stable hybrid myotubes, , European journal of histochemistry : EJH, 1993
A light and electron microscopic study of dystrophin localization at the mouse neuromuscular junction, , Synapse (New York, N.Y.), 1992, 10.1002/syn.890100202
Emerging Perspectives on Prime Editor Delivery to the Brain, Jacques P. Tremblay, Marc Lavertu, Avi Schroeder, Joël Rousseau, Yaoyao Lu, Sina Ramezanian, Eli BenDavid, Pharmaceuticals, 2024, 10.3390/ph17060763
Stem and progenitor cells in skeletal muscle development, maintenance, and therapy, , Molecular therapy : the journal of the American Society of Gene Therapy, 2007, 10.1038/mt.sj.6300145
Current Clinical Applications of In Vivo Gene Therapy with AAVs, , Molecular therapy : the journal of the American Society of Gene Therapy, 2021, 10.1016/j.ymthe.2020.12.007
Intramuscular Transplantation of Muscle Precursor Cells over-expressing MMP-9 improves Transplantation Success, , PLoS currents, 2011, 10.1371/currents.RRN1275
Base editing strategy for insertion of the A673T mutation in the APP gene to prevent the development of AD in vitro, , Molecular therapy. Nucleic acids, 2021, 10.1016/j.omtn.2021.02.032
Finding an Appropriate Mouse Model to Study the Impact of a Treatment for Friedreich Ataxia on the Behavioral Phenotype, Jacques P. Tremblay, Vanessa Couture, Pouiré Yameogo, Gabrielle Buisson, Malek Aloui, Nathalie Majeau, Solange Gni-fiene Yanyabé, Catherine Gérard, Camille Bouchard, Genes, 2023, 10.3390/genes14081654
Baclofen modifies via the release of monoamines the synaptic depression, frequency facilitation, and posttetanic potentiation observed at an identified cholinergic synapse of Aplysia californica, , Canadian journal of physiology and pharmacology, 1981, 10.1139/y81-039
Myoblast transplantation: a brief review of the problems and of some solutions, , Basic and applied myology : BAM, 1997
Immunosuppression with monoclonal antibodies and CTLA4-Ig after myoblast transplantation in mice, , Transplantation, 1996, 10.1097/00007890-199610150-00015
The effects of hemicholinium-3 on synaptic depression, facilitation, and post-tetanic potentiation at an identified synapse of Aplysia californica, , Canadian journal of physiology and pharmacology, 1980, 10.1139/y80-064
Increased interferon-gamma mRNA expression following alloincompatible myoblast transplantation is inhibited by FK506, , Muscle & nerve, 1996, 10.1002/(SICI)1097-4598(199607)19:73.0.CO;2-B
Endonucleases: tools to correct the dystrophin gene, , The journal of gene medicine, 2011, 10.1002/jgm.1611
Efficient Restoration of the Dystrophin Gene Reading Frame and Protein Structure in DMD Myoblasts Using the CinDel Method, , Molecular therapy. Nucleic acids, 2016, 10.1038/mtna.2015.58
Sarcolemmal Complement Membrane Attack Complex Deposits During Acute Rejection of Myofibers in Nonhuman Primates, , Journal of neuropathology and experimental neurology, 2019, 10.1093/jnen/nly106
The urokinase plasminogen activator: an interesting way to improve myoblast migration following their transplantation, , Experimental cell research, 2002, 10.1006/excr.2002.5642
Transplantation of normal or genetically modified myoblasts for the treatment of hereditary or acquired diseases, , Journal de la Societe de biologie, 2001
First study of intra-arterial delivery of myogenic mononuclear cells to skeletal muscles in primates, , Cell transplantation, 2014, 10.3727/096368914X685032
Online Manuscript Processing for Cell Transplantation, , Cell transplantation, 2006, 10.3727/000000006783981738
Transplantation of human myoblasts in SCID mice as a potential muscular model for myotonic dystrophy, , Journal of neuropathology and experimental neurology, 1999, 10.1097/00005072-199909000-00003
Benzodiazepines modify synaptic depression, frequency facilitation and PTP an identified cholinergic synapse of Aplysia, , Life sciences, 1980, 10.1016/0024-3205(80)90130-7
Postsynaptic structure may account for variations in miniature endplate current shapes along frog neuromuscular junctions, , Synapse (New York, N.Y.), 1990, 10.1002/syn.890050402
Telomerase allows the immortalization of T antigen-positive DMD myoblasts: a new source of cells for gene transfer application, , Gene therapy, 2000, 10.1038/sj.gt.3301132
Myogenesis: where and how can we get skeletal myoblasts?, , Dialogues in cardiovascular medicine : DCM, 2003
Exercise improves the success of myoblast transplantation in mdx mice, , Neuromuscular disorders : NMD, 2006, 10.1016/j.nmd.2006.06.003
High efficiency of muscle regeneration after human myoblast clone transplantation in SCID mice, , The Journal of clinical investigation, 1994, 10.1172/JCI117011
Male-specific competitive PCR for the quantification of transplanted cells in mice, , BioTechniques, 1999, 10.2144/99273bm05
Therapeutic effects of exon skipping and losartan on skeletal muscle of mdx mice, , Pathology international, 2014, 10.1111/pin.12190
Losartan enhances the success of myoblast transplantation, , Cell transplantation, 2012, 10.3727/096368911X576045
Development and characterization of a simian virus 40 immortalized bovine endometrial stromal cell line, , Endocrinology, 2009, 10.1210/en.2008-0744
Modulation of synapse RC1-R15 of Aplysia californica by fiber(s) of the right connective, , Canadian journal of physiology and pharmacology, 1982, 10.1139/y82-094
Myoblast transplantation in skeletal muscles, , 2012
Cell therapies for inherited myopathies, , Current opinion in rheumatology, 2003, 10.1097/00002281-200311000-00007
Dystrophin expression in host muscle following transplantation of muscle precursor cells modified with the phiC31 integrase, , Gene therapy, 2007, 10.1038/sj.gt.3302887
CRISPR-Cas9 Gene Therapy for Duchenne Muscular Dystrophy, , Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics, 2022, 10.1007/s13311-022-01197-9
Cyclophosphamide immunosuppression does not permit successful myoblast allotransplantation in mouse, , Neuromuscular disorders : NMD, 1995, 10.1016/0960-8966(95)00011-b
Evaluation of various gene transfection methods into human myoblast clones, , Transplantation proceedings, 1992
Human myoblast transplantation between immunohistocompatible donors and recipients produces immune reactions, , Transplantation proceedings, 1992
Immunosuppression with FK 506 insures good success of myoblast transplantation in MDX mice, , Transplantation proceedings, 1994
CRISPR/Cas9 delivery strategies with engineered extracellular vesicles, Jacques P. Tremblay, Gabriel Lamothe, Kelly Godbout, Yaoyao Lu, Molecular Therapy - Nucleic Acids, 2023, 10.1016/j.omtn.2023.102040
Miniature endplate potential amplitudes corrected for spatial decay are not normally distributed, , Brain research, 1985, 10.1016/0006-8993(85)91339-3
Increased Frataxin Expression Induced in Friedreich Ataxia Cells by Platinum TALE-VP64s or Platinum TALE-SunTag, , Molecular therapy. Nucleic acids, 2018, 10.1016/j.omtn.2018.04.009
Non-uniform responses to Ca2+ along the frog neuromuscular junction: effects on the probability of spontaneous and evoked transmitter release, , Neuroscience, 1991, 10.1016/0306-4522(91)90142-b
CRISPR-induced deletion with SaCas9 restores dystrophin expression in dystrophic models in vitro and in vivo, , 2018, 10.1101/378331
Myoblast transplantation between monozygotic twin girl carriers of Duchenne muscular dystrophy, , Neuromuscular disorders : NMD, 1993, 10.1016/0960-8966(93)90121-y
CRISPR-Induced Deletion with SaCas9 Restores Dystrophin Expression in Dystrophic Models In Vitro and In Vivo, , Molecular therapy : the journal of the American Society of Gene Therapy, 2018, 10.1016/j.ymthe.2018.08.010
A first semimanual device for clinical intramuscular repetitive cell injections, , Cell transplantation, 2010, 10.3727/096368909X478812
Resting and stimulated values of model parameters governing transmitter release at a synapse in Aplysia californica, , Brain research, 1976, 10.1016/0006-8993(76)90378-4
In vivo stimulation of a cholinergic synapse of the chick ciliary ganglion induces a reduction in the number of dense core vesicles, , Neuroscience letters, 1981, 10.1016/0304-3940(81)90176-2
Myoblast allotransplantation in mice: degree of success varies depending on the efficacy of various immunosuppressive treatments, , Transplantation proceedings, 1994
Presynaptic modulating effects of GABA on depression, facilitation, and posttetanic potentiation of a cholinergic synapse in Aplysia californica, , Canadian journal of physiology and pharmacology, 1977, 10.1139/y77-174
Intraarterial injection of muscle-derived CD34(+)Sca-1(+) stem cells restores dystrophin in mdx mice, , The Journal of cell biology, 2001, 10.1083/jcb.152.2.335
Growth factor coinjection improves the migration potential of monkey myogenic precursors without affecting cell transplantation success, , Cell transplantation, 2009, 10.3727/096368909X470900
Growth factors improve the in vivo migration of human skeletal myoblasts by modulating their endogenous proteolytic activity, , Transplantation, 2004, 10.1097/01.tp.0000131175.60047.eb
Development of a web course on gene therapy by the international consortium of gene therapy, , Molecular therapy : the journal of the American Society of Gene Therapy, 2014, 10.1038/mt.2014.11
Resetting the problem of cell death following muscle-derived cell transplantation: detection, dynamics and mechanisms, , Journal of neuropathology and experimental neurology, 2003, 10.1093/jnen/62.9.951
Transplanted myoblasts can migrate several millimeters to fuse with damaged myofibers in nonhuman primate skeletal muscle, , Journal of neuropathology and experimental neurology, 2011, 10.1097/NEN.0b013e31822a6baa
Frequency facilitation and post-tetanic potentiation of a unitary synaptic potential in Aplysia californica are limited by different processes, , Brain research, 1976, 10.1016/0006-8993(76)90377-2
Implantation of myogenic cells in skeletal muscles, , 2010
Synaptic contacts on glial cells in the abdominal ganglion of Aplysia californica, , The Journal of comparative neurology, 1979, 10.1002/cne.901880304
Increased Expression of Laminin Subunit Alpha 1 Chain by dCas9-VP160, , Molecular therapy. Nucleic acids, 2017, 10.1016/j.omtn.2016.11.004
Heterogeneous labeling of mitochondrial membrane, endoplasmic reticulum, neurofilaments and synaptic vesicles with a lectin (DBA-peroxidase), , Neuroscience letters, 1982, 10.1016/0304-3940(82)90003-9
Past, present and future of myoblast transplantation in the treatment of Duchenne muscular dystrophy, , Pediatric transplantation, 2010, 10.1111/j.1399-3046.2010.01377.x
Reduction of postjunctional fold density and depth in dystrophic mice, , Synapse (New York, N.Y.), 1988, 10.1002/syn.890020206
Tumor necrosis factor-alpha (TNF-alpha) stimulates chemotactic response in mouse myogenic cells, , Cell transplantation, 2003, 10.3727/000000003783985115
Myoblast transplantation: techniques in nonhuman primates as a bridge to clinical trials, , 2011
A monoclonal antibody to beta-tubulin distinguishes a subset of neurons and axons in the chick ciliary ganglion, , Canadian journal of biochemistry and cell biology = Revue canadienne de biochimie et biologie cellulaire, 1985, 10.1139/o85-065
Expression of major histocompatibility complex antigens on human myoblasts, , Transplantation proceedings, 1991
Meganucleases can restore the reading frame of a mutated dystrophin, , Gene therapy, 2010, 10.1038/gt.2010.26
Necrosis, sarcolemmal damage and apoptotic events in myofibers rejected by CD8+ lymphocytes: Observations in nonhuman primates, , Neuromuscular disorders : NMD, 2012, 10.1016/j.nmd.2012.05.005
Translating the genomics revolution: the need for an international gene therapy consortium for monogenic diseases, , Molecular therapy : the journal of the American Society of Gene Therapy, 2013, 10.1038/mt.2013.4
A case for immunosuppression for myoblast transplantation in duchenne muscular dystrophy, , Molecular therapy : the journal of the American Society of Gene Therapy, 2009, 10.1038/mt.2009.125
Prior culture with concanavalin A increases intramuscular migration of transplanted myoblast, , Muscle & nerve, 1998, 10.1002/(sici)1097-4598(199803)21:33.0.co;2-5
First test of a high-density injection protocol for myogenic cell transplantation throughout large volumes of muscles in a Duchenne muscular dystrophy patient: eighteen months follow-up, , Neuromuscular disorders : NMD, 2007, 10.1016/j.nmd.2006.10.003
Transfection of large plasmids in primary human myoblasts, , Gene therapy, 2001, 10.1038/sj.gt.3301532
Prevention by anti-LFA-1 of acute myoblast death following transplantation, , Journal of immunology (Baltimore, Md. : 1950), 1997
The Process of Engraftment of Myogenic Cells in Skeletal Muscles of Primates: Understanding Clinical Observations and Setting Directions in Cell Transplantation Research, , Cell transplantation, 2017, 10.1177/0963689717724798
Approche Thérapeutique de la myopathie de Duchenne par transplantation de myoblastes, , Medecine sciences : M/S, 1991
Percutaneous versus surgical delivery of autologous myoblasts after chronic myocardial infarction: an in vivo cardiovascular magnetic resonance study, , Catheterization and cardiovascular interventions : official journal of the Society for Cardiac Angiography & Interventions, 2010, 10.1002/ccd.22204
Thérapie cellulaire et génique, , 2015
Administration of a soluble activin type IIB receptor promotes the transplantation of human myoblasts in dystrophic mice, , Cell transplantation, 2012, 10.3727/096368911X627480
Human myoblast transplantation: preliminary results of 4 cases, , Muscle & nerve, 1992, 10.1002/mus.880150504
Enzymatic activity and morphological differentiation in de novo innervated human muscle cultures, , European journal of histochemistry : EJH, 1994
Increased granzyme B mRNA after alloincompatible myoblast transplantation, , Transplantation, 1995
Frequency and amplitude gradients of spontaneous release along the length of the frog neuromuscular junction, , Synapse (New York, N.Y.), 1989, 10.1002/syn.890030402
Dynamics of the early immune cellular reactions after myogenic cell transplantation, , Cell transplantation, 2002, 10.3727/000000002783985378
Generation of lentiviral vectors for use in skeletal muscle research, , Methods in molecular biology (Clifton, N.J.), 2012, 10.1007/978-1-61779-343-1_16
Myoblast transplantation: a possible surgical treatment for a severe pediatric disease, , Surgery today, 2010, 10.1007/s00595-009-4242-z
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Preservation of muscle spindles in a 27-year-old Duchenne muscular dystrophy patient: importance for regenerative medicine strategies, , Muscle & nerve, 2010, 10.1002/mus.21644
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Human myoblast transplantation: a simple assay for tumorigenicity, , Neuromuscular disorders : NMD, 1991, 10.1016/0960-8966(91)90120-h
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Myotubes Formed De Novo by Myoblasts Injected into the Scar of Myocardial Infarction Persisted for 16 Years in a Patient: Importance for Regenerative Medicine in Degenerative Myopathies, , Stem cells translational medicine, 2019, 10.1002/sctm.18-0202
Myoblast transplantation in non-dystrophic dog, , Neuromuscular disorders : NMD, 1998, 10.1016/s0960-8966(97)00148-x
Systemic production of human granulocyte colony-stimulating factor in nonhuman primates by transplantation of genetically modified myoblasts, , Human gene therapy, 2000, 10.1089/10430340050032384
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An AAV9 coding for frataxin clearly improved the symptoms and prolonged the life of Friedreich ataxia mouse models, , Molecular therapy. Methods & clinical development, 2014, 10.1038/mtm.2014.44
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A Historic Recapitulation of Myoblast Transplantation, , 2006
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Gene Therapy in Duchenne’s Muscular Dystrophy, , 2009
Mechanism of increasing dystrophin-positive myofibers by myoblast transplantation: study using mdx/beta-galactosidase transgenic mice, , Acta neuropathologica, 1996, 10.1007/s004010050456
Inhibiting myostatin with follistatin improves the success of myoblast transplantation in dystrophic mice, , Cell transplantation, 2008, 10.3727/096368908784153913
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A synthetic mechano growth factor E Peptide enhances myogenic precursor cell transplantation success, , American journal of transplantation : official journal of the American Society of Transplantation and the American Society of Transplant Surgeons, 2007, 10.1111/j.1600-6143.2007.01927.x
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Successive injections in mdx mice of myoblasts grown with bFGF, , Neuromuscular disorders : NMD, 1996, 10.1016/0960-8966(96)00004-1
Assessment of a symptomatic Duchenne muscular dystrophy carrier 20 years after myoblast transplantation from her asymptomatic identical twin sister, , Neuromuscular disorders : NMD, 2013, 10.1016/j.nmd.2013.04.007
Development of an AAV9 coding for a 3XFLAG-TALEfrat#8-VP64 able to increase in vivo the human frataxin in YG8R mice, , Gene therapy, 2016, 10.1038/gt.2016.36
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De Novo Circulating Antidonor's Cell Antibodies During Induced Acute Rejection of Allogeneic Myofibers in Myogenic Cell Transplantation: A Study in Nonhuman Primates, , Transplantation direct, 2017, 10.1097/TXD.0000000000000740
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From gRNA Identification to the Restoration of Dystrophin Expression: A Dystrophin Gene Correction Strategy for Duchenne Muscular Dystrophy Mutations Using the CRISPR-Induced Deletion Method, , Methods in molecular biology (Clifton, N.J.), 2018, 10.1007/978-1-4939-7374-3_19
Removal of the GAA repeat in the heart of a Friedreich’s ataxia mouse model using CjCas9, Jacques P. Tremblay, Nathalie Majeau, Catherine Gérard, Pouiré Yaméogo, Gene Therapy, 2023, 10.1038/s41434-023-00387-0
Use of fluorescent latex microspheres (FLMs) to follow the fate of transplanted myoblasts, , The journal of histochemistry and cytochemistry : official journal of the Histochemistry Society, 1993, 10.1177/41.10.8245416
Blocking the myostatin signal with a dominant negative receptor improves the success of human myoblast transplantation in dystrophic mice, , Molecular therapy : the journal of the American Society of Gene Therapy, 2011, 10.1038/mt.2010.171
Myogenic progenitor cells derived from human induced pluripotent stem cell are immune-tolerated in humanized mice, , Stem cells translational medicine, 2021, 10.1002/sctm.19-0452
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Experimental and therapeutic approaches to muscular dystrophies, , Current opinion in neurology, 2002, 10.1097/00019052-200210000-00007
A new pro-migratory activity on human myogenic precursor cells for a synthetic peptide within the E domain of the mechano growth factor, , Experimental cell research, 2007, 10.1016/j.yexcr.2006.10.032
Electrotransfer of naked DNA in the skeletal muscles of animal models of muscular dystrophies, , Gene therapy, 2001, 10.1038/sj.gt.3301484
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Therapeutic Strategies for Dystrophin Replacement in Duchenne Muscular Dystrophy, , Frontiers in medicine, 2022, 10.3389/fmed.2022.859930
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Rapid selection of donor myoblast clones for muscular dystrophy therapy using cell surface expression of NCAM, , European journal of histochemistry : EJH, 1993
1,25-dihydroxyvitamin D3 increases the transplantation success of human muscle precursor cells in SCID mice, , Cell transplantation, 2007, 10.3727/000000007783464876
Myoblast transplantation in monkeys: control of immune response by FK506, , Journal of neuropathology and experimental neurology, 1996, 10.1097/00005072-199606000-00002
Evidence of mdx mouse skeletal muscle fragility in vivo by eccentric running exercise, , Muscle & nerve, 1998, 10.1002/(sici)1097-4598(199805)21:53.0.co;2-6
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Serum Extracellular Vesicles for Delivery of Crispr-Cas9 Ribonucleoproteins to Modify the Dystrophin Gene, , Molecular therapy : the journal of the American Society of Gene Therapy, 2022, 10.1016/j.ymthe.2022.05.023
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Gene transfer using HACs: a key step closer to ex vivo gene therapy using autologous gene-corrected cells to treat muscular dystrophy, , Molecular therapy : the journal of the American Society of Gene Therapy, 2011, 10.1038/mt.2011.254
Successful myoblast transplantation in fibrotic muscles: no increased impairment by the connective tissue, , Transplantation, 1999, 10.1097/00007890-199906270-00018
Induction of Anoikis following myoblast transplantation into SCID mouse muscles requires the Bit1 and FADD pathways, , American journal of transplantation : official journal of the American Society of Transplantation and the American Society of Transplant Surgeons, 2007, 10.1111/j.1600-6143.2007.01830.x
Limb–Girdle Muscular Dystrophies Classification and Therapies, Jacques P. Tremblay, Camille Bouchard, Journal of Clinical Medicine, 2023, 10.3390/jcm12144769
Effects of vitamin C on cytotherapy-mediated muscle regeneration, , Cell transplantation, 2013, 10.3727/096368912X657846
Rational design of a serum-free culture medium for the growth of human myoblasts destined to cell therapy, , The Canadian journal of chemical engineering, 2016, 10.1002/cjce.22586
Autotransplantation in mdx mice of mdx myoblasts genetically corrected by an HSV-1 amplicon vector, , Cell transplantation, 2002
Sensorimotor skills in Fxn KO/Mck mutants deficient for frataxin in muscle, , Brain research, 2015, 10.1016/j.brainres.2015.03.001
Meeting of the Cell Transplantation Society in Miami, , Cell transplantation, 1997
Nonfunctional mutant Wrn protein leads to neurological deficits, neuronal stress, microglial alteration, and immune imbalance in a mouse model of Werner syndrome, , Brain, behavior, and immunity, 2018, 10.1016/j.bbi.2018.06.007
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Myoblasts derived from normal hESCs and dystrophic hiPSCs efficiently fuse with existing muscle fibers following transplantation, , Molecular therapy : the journal of the American Society of Gene Therapy, 2012, 10.1038/mt.2012.188
Three Decades of Clinical Gene Therapy: From Experimental Technologies to Viable Treatments, , Molecular therapy : the journal of the American Society of Gene Therapy, 2021, 10.1016/j.ymthe.2021.01.013
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Results of a triple blind clinical study of myoblast transplantations without immunosuppressive treatment in young boys with Duchenne muscular dystrophy, , Cell transplantation, 1993, 10.1177/096368979300200203
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Cytomegalovirus and myoblast transplantation, , Lancet (London, England), 1991, 10.1016/0140-6736(91)93090-v
CRISPR-SCReT (CRISPR-Stop Codon Read Through) method to control Cas9 expression for gene editing, , Gene therapy, 2022, 10.1038/s41434-021-00297-z
Tubulyzine, a novel tri-substituted triazine, prevents the early cell death of transplanted myogenic cells and improves transplantation success, , Biochemistry and cell biology = Biochimie et biologie cellulaire, 2003, 10.1139/o03-054
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Gene Editing for Duchenne Muscular Dystrophy Using the CRISPR/Cas9 Technology: The Importance of Fine-tuning the Approach, , Molecular therapy : the journal of the American Society of Gene Therapy, 2016, 10.1038/mt.2016.191
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In Vitro Correction of Point Mutations in the DYSF Gene Using Prime Editing, Jacques P. Tremblay, Sina Ramezani, Laura Bastrenta, Marie Dubost, Gabriel Lamothe, Joël Rousseau, Camille Bouchard, International Journal of Molecular Sciences, 2025, 10.3390/ijms26125647
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Insertion of the Icelandic Mutation (A673T) by Prime Editing: A Potential Preventive Treatment for Familial and Sporadic Alzheimer's Disease, , The CRISPR journal, 2022, 10.1089/crispr.2021.0085
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Characterization of frataxin gene network in Friedreich's ataxia fibroblasts using the RNA-Seq technique, , Mitochondrion, 2016, 10.1016/j.mito.2016.06.003
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Novel Duchenne muscular dystrophy treatment through myoblast transplantation tolerance with anti-CD45RB, anti-CD154 and mixed chimerism, , American journal of transplantation : official journal of the American Society of Transplantation and the American Society of Transplant Surgeons, 2004, 10.1111/j.1600-6143.2004.00501.x
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