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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 T1709M 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

Cerebroside may be falsely identified as a soluble 'brain specific protein', , Journal of neurochemistry, 1974, 10.1111/j.1471-4159.1974.tb04360.x
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
Intramuscular transplantation of myogenic cells in primates: importance of needle size, cell number, and injection volume, , Cell transplantation, 2014, 10.3727/096368912X661337
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
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
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
Fast turnover of clear vesicle membrane in frog sympathetic ganglion, , Neuroscience, 1986, 10.1016/0306-4522(86)90093-x
Myoblast transplantation in skeletal muscles, , 2012
Is dystrophin present in the nerve terminal at the neuromuscular junction? An immunohistochemical study of the heterozygote dystrophic (mdx) mouse, , Synapse (New York, N.Y.), 1991, 10.1002/syn.890070207
Therapeutic Strategies for Dystrophin Replacement in Duchenne Muscular Dystrophy, , Frontiers in medicine, 2022, 10.3389/fmed.2022.859930
A Historic Recapitulation of Myoblast Transplantation, , 2006
Evaluation of various gene transfection methods into human myoblast clones, , Transplantation proceedings, 1992
Myoblast transplantation: a brief review of the problems and of some solutions, , Basic and applied myology : BAM, 1997
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
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
Inhibiting myostatin with follistatin improves the success of myoblast transplantation in dystrophic mice, , Cell transplantation, 2008, 10.3727/096368908784153913
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
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
Transfection of large plasmids in primary human myoblasts, , Gene therapy, 2001, 10.1038/sj.gt.3301532
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
The advances and challenges of Gene Therapy for Duchenne Muscular Dystrophy, , Journal of Genetic Medicine and Gene Therapy, 2017, 10.29328/journal.jgmgt.1001003
Autotransplantation in mdx mice of mdx myoblasts genetically corrected by an HSV-1 amplicon vector, , Cell transplantation, 2002
Endonucleases: tools to correct the dystrophin gene, , The journal of gene medicine, 2011, 10.1002/jgm.1611
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
Fibrin gel improves the survival of transplanted myoblasts, , Cell transplantation, 2012, 10.3727/096368911X576018
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
Treatment with anti-CD154 antibody and donor-specific transfusion prevents acute rejection of myoblast transplantation, , Transplantation, 2002, 10.1097/00007890-200202150-00021
Benzodiazepines modify synaptic depression, frequency facilitation and PTP an identified cholinergic synapse of Aplysia, , Life sciences, 1980, 10.1016/0024-3205(80)90130-7
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
A first semimanual device for clinical intramuscular repetitive cell injections, , Cell transplantation, 2010, 10.3727/096368909X478812
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
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
Miniature endplate potential amplitudes corrected for spatial decay are not normally distributed, , Brain research, 1985, 10.1016/0006-8993(85)91339-3
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
The first Joint Conference of the Cell Transplant Society (CTS), International Pancreas and Islet Transplant Association (IPITA), and International Xenotransplantation Association (IXA), all sections of The Transplantation Society (TTS), took place in Minneapolis, Minn, USA, from September 15-20, 2007. Preface, , Transplantation proceedings, 2008, 10.1016/j.transproceed.2008.02.021
Dysferlin expression after normal myoblast transplantation in SCID and in SJL mice, , Neuromuscular disorders : NMD, 2002, 10.1016/s0960-8966(01)00254-1
Tumor necrosis factor-alpha (TNF-alpha) stimulates chemotactic response in mouse myogenic cells, , Cell transplantation, 2003, 10.3727/000000003783985115
Human myoblast transplantation in immunodeficient and immunosuppressed mice: evidence of rejection, , Muscle & nerve, 1994, 10.1002/mus.880170214
Myoblast transplantation in whole muscle of nonhuman primates, , Journal of neuropathology and experimental neurology, 2000, 10.1093/jnen/59.3.197
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
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
Prevention by anti-LFA-1 of acute myoblast death following transplantation, , Journal of immunology (Baltimore, Md. : 1950), 1997
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
Dystrophin expression in myofibers of Duchenne muscular dystrophy patients following intramuscular injections of normal myogenic cells, , Molecular therapy : the journal of the American Society of Gene Therapy, 2004, 10.1016/j.ymthe.2003.11.023
Increased granzyme B mRNA after alloincompatible myoblast transplantation, , Transplantation, 1995
Growth factor coinjection improves the migration potential of monkey myogenic precursors without affecting cell transplantation success, , Cell transplantation, 2009, 10.3727/096368909X470900
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
Dystrophin expression in myotubes formed by the fusion of normal and dystrophic myoblasts, , Muscle & nerve, 1991, 10.1002/mus.880140213
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
Scanning electron microscopic study of the neuromuscular junction of dystrophic mice, , Experimental neurology, 1988, 10.1016/0014-4886(88)90200-2
Transplantation of normal or genetically modified myoblasts for the treatment of hereditary or acquired diseases, , Journal de la Societe de biologie, 2001
Systemic production of human granulocyte colony-stimulating factor in nonhuman primates by transplantation of genetically modified myoblasts, , Human gene therapy, 2000, 10.1089/10430340050032384
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 Reiken, Mathieu Dugas, Kelly Godbout, International Journal of Molecular Sciences, 2025, 10.3390/ijms26072835
Another new super muscle stem cell leaves unaddressed the real problems of cell therapy for duchenne muscular dystrophy, , Molecular therapy : the journal of the American Society of Gene Therapy, 2008, 10.1038/mt.2008.243
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
Immunosuppression with monoclonal antibodies and CTLA4-Ig after myoblast transplantation in mice, , Transplantation, 1996, 10.1097/00007890-199610150-00015
Mechanism of increasing dystrophin-positive myofibers by myoblast transplantation: study using mdx/beta-galactosidase transgenic mice, , Acta neuropathologica, 1996, 10.1007/s004010050456
Deletion of the GAA repeats from the human frataxin gene using the CRISPR-Cas9 system in YG8R-derived cells and mouse models of Friedreich ataxia, , Gene therapy, 2017, 10.1038/gt.2016.89
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
Portrait of Dysferlinopathy: Diagnosis and Development of Therapy, Jacques P. Tremblay, Camille Bouchard, Journal of Clinical Medicine, 2023, 10.3390/jcm12186011
Myoblast transplantation: the current status of a potential therapeutic tool for myopathies, , Journal of muscle research and cell motility, 2003
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
Cytomegalovirus and myoblast transplantation, , Lancet (London, England), 1991, 10.1016/0140-6736(91)93090-v
Cholinergic agents affect two receptors that modulate transmitter release at a central synapse in Aplsia californica, , Brain research, 1975, 10.1016/0006-8993(75)90657-5
Intramuscular Transplantation of Muscle Precursor Cells over-expressing MMP-9 improves Transplantation Success, , PLoS currents, 2011, 10.1371/currents.RRN1275
Successful myoblast transplantation in fibrotic muscles: no increased impairment by the connective tissue, , Transplantation, 1999, 10.1097/00007890-199906270-00018
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
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
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
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
Myogenesis: where and how can we get skeletal myoblasts?, , Dialogues in cardiovascular medicine : DCM, 2003
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
Cell therapy in muscular dystrophies: many promises in mice and dogs, few facts in patients, , Expert opinion on biological therapy, 2015, 10.1517/14712598.2015.1057564
Immune reactions after myoblast transplantation in mouse muscles, , Transplantation proceedings, 1992
Characterization of frataxin gene network in Friedreich's ataxia fibroblasts using the RNA-Seq technique, , Mitochondrion, 2016, 10.1016/j.mito.2016.06.003
Myoblast allotransplantation in mice: degree of success varies depending on the efficacy of various immunosuppressive treatments, , Transplantation proceedings, 1994
Improved success of myoblast transplantation in mdx mice by blocking the myostatin signal, , Transplantation, 2005, 10.1097/01.tp.0000167379.27872.2b
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
Advances & challenges of using CRISPR-Cas9 gene editing for treating Duchenne muscular dystrophy, , Cell & gene therapy insights, 2017, 10.18609/cgti.2017.003
Myocardial regeneration : which cell and why, , 2006
Non monotonic morphometric changes produced at mouse neuromuscular junctions following in vivo stimulation at various frequencies, , Experimental brain research, 1986, 10.1007/BF00237590
Successive injections in mdx mice of myoblasts grown with bFGF, , Neuromuscular disorders : NMD, 1996, 10.1016/0960-8966(96)00004-1
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
Combined immunosuppression of mycophenolate mofetil and FK506 for myoblast transplantation in mdx mice, , Transplantation, 2001, 10.1097/00007890-200107150-00010
In vivo migration of transplanted myoblasts requires matrix metalloproteinase activity, , Experimental cell research, 2000, 10.1006/excr.2000.4962
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
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
Laminin-111: a potential therapeutic agent for Duchenne muscular dystrophy, , Molecular therapy : the journal of the American Society of Gene Therapy, 2010, 10.1038/mt.2010.165
Expression of human dystrophin following the transplantation of genetically modified mdx myoblasts, , Gene therapy, 1998, 10.1038/sj.gt.3300753
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
Lymphocyte infiltration following allo- and xenomyoblast transplantation in mdx mice, , Muscle & nerve, 1995, 10.1002/mus.880180107
Heterosynaptic inhibition modifies the presynaptic plasticities of the transmission process at the synapse in Aplysia californica, , Brain research, 1976, 10.1016/0006-8993(76)90381-4
Prime Editing for Human Gene Therapy: Where Are We Now?, Jacques P. Tremblay, Kelly Godbout, Cells, 2023, 10.3390/cells12040536
Meganucleases can restore the reading frame of a mutated dystrophin, , Gene therapy, 2010, 10.1038/gt.2010.26
RCAS1 is associated with ductal breast cancer progression, , Biochemical and biophysical research communications, 2002, 10.1016/S0006-291X(02)00401-1
Gaba and benzodiazepines in invertebrate species, , 1988
A new technique to identify hybrid myotubes in vitro without culture fixation, , The journal of histochemistry and cytochemistry : official journal of the Histochemistry Society, 1991, 10.1177/39.1.1701185
1,25-dihydroxyvitamin D3 increases the transplantation success of human muscle precursor cells in SCID mice, , Cell transplantation, 2007, 10.3727/000000007783464876
Interleukin-4 improves the migration of human myogenic precursor cells in vitro and in vivo, , Experimental cell research, 2006, 10.1016/j.yexcr.2006.01.002
AG490 improves the survival of human myoblasts in vitro and in vivo, , Cell transplantation, 2012, 10.3727/096368912X655028
Losartan enhances the success of myoblast transplantation, , Cell transplantation, 2012, 10.3727/096368911X576045
Effects of vitamin C on cytotherapy-mediated muscle regeneration, , Cell transplantation, 2013, 10.3727/096368912X657846
CSCI/RCPSC Henry Friesen Lecture: Cell therapy for Duchenne muscular dystrophy, , Clinical and investigative medicine. Medecine clinique et experimentale, 2006
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
Successful myoblast allotransplantation in mdx mice using rapamycin, , Transplantation, 1995
Myoblast transplantation produced dystrophin-positive muscle fibres in a 16-year-old patient with Duchenne muscular dystrophy, , Clinical science (London, England : 1979), 1991, 10.1042/cs0810287
Functional EGFP-dystrophin fusion proteins for gene therapy vector development, , Protein engineering, 2000, 10.1093/protein/13.9.611
Enzymatic activity and morphological differentiation in de novo innervated human muscle cultures, , European journal of histochemistry : EJH, 1994
Muscle glucose-6-phosphate dehydrogenase deficiency: restoration of enzymatic activity in hybrid myotubes, , Muscle & nerve, 1993, 10.1002/mus.880160604
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
Interrelation between MEPP amplitude and MEPP frequency in different regions along the frog neuromuscular junction, , Brain research, 1987, 10.1016/0006-8993(87)90404-5
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
Base editing strategy allows insertion of the A673T mutation in APP gene to prevent the development of Alzheimer’s disease, Jacques P. Tremblay, Gabriel Lamothe, Tom Bertin, Francis-Gabriel Bégin, Joël Rousseau, Antoine Guyon, 2020, 10.1101/2020.10.27.357830
Use of normal and genetically modified myoblasts for the treatment of myopathies, , 2000
Transplantation of dermal fibroblasts expressing MyoD1 in mouse muscles, , Biochemical and biophysical research communications, 1998, 10.1006/bbrc.1998.8995
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
Exercise improves the success of myoblast transplantation in mdx mice, , Neuromuscular disorders : NMD, 2006, 10.1016/j.nmd.2006.06.003
Myoblast transplantation: a possible surgical treatment for a severe pediatric disease, , Surgery today, 2010, 10.1007/s00595-009-4242-z
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
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
Cytoplasmic restoration and persistence of glucose-6-phosphate dehydrogenase activity in stable hybrid myotubes, , European journal of histochemistry : EJH, 1993
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
Role of non-major histocompatibility complex antigens in the rejection of transplanted myoblasts, , Transplantation, 1997, 10.1097/00007890-199703270-00016
Immune Reaction Following Cell and Gene Therapy, , 2000
Experimental and therapeutic approaches to muscular dystrophies, , Current opinion in neurology, 2002, 10.1097/00019052-200210000-00007
Intramuscular migration of myoblasts transplanted after muscle pretreatment with metalloproteinases, , Cell transplantation, 2000, 10.1177/096368970000900410
Polymerase chain reaction-based assay to assess the success of myoblast transplantation in mdx mice, , Transplantation proceedings, 1994
Control of inflammatory damage by anti-LFA-1: increase success of myoblast transplantation, , Cell transplantation, 1997
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
Electrotransfer of naked DNA in the skeletal muscles of animal models of muscular dystrophies, , Gene therapy, 2001, 10.1038/sj.gt.3301484
The CRISPR system can correct or modify the expression of genes responsible for hereditary diseases, , Medecine sciences : M/S, 2015, 10.1051/medsci/20153111016
Development and characterization of a simian virus 40 immortalized bovine endometrial stromal cell line, , Endocrinology, 2009, 10.1210/en.2008-0744
Antibody formation after myoblast transplantation in Duchenne-dystrophic patients, donor HLA compatible, , Transplantation proceedings, 1993
Section VI - Novel Genetic Technologies. 6.2 Gene Therapy, , 2009
Increased number per area of peptidergic and cholinergic vesicles in synapses of the chick ciliary ganglion following 10 Hz in vivo stimulation, , Neuroscience letters, 1983, 10.1016/0304-3940(83)90542-6
Gene therapy: a strategy for the treatment of inherited muscle diseases?, , Current opinion in pharmacology, 2001, 10.1016/s1471-4892(01)00052-2
Intracellular delivery of a Tat-eGFP fusion protein into muscle cells, , Molecular therapy : the journal of the American Society of Gene Therapy, 2001, 10.1006/mthe.2001.0279
Rapid selection of donor myoblast clones for muscular dystrophy therapy using cell surface expression of NCAM, , European journal of histochemistry : EJH, 1993
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
High efficiency of muscle regeneration after human myoblast clone transplantation in SCID mice, , The Journal of clinical investigation, 1994, 10.1172/JCI117011
Early and massive death of myoblasts transplanted into skeletal muscle : responsible factors and potential solutions, , Current opinion in organ transplantation, 2007
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
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
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
Efficient myoblast transplantation in mice immunosuppressed with monoclonal antibodies and CTLA4 Ig, , Transplantation proceedings, 1997, 10.1016/s0041-1345(97)00166-8
Very efficient myoblast allotransplantation in mice under FK506 immunosuppression, , Muscle & nerve, 1994, 10.1002/mus.880171210
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
Distribution of spontaneous release along the frog neuromuscular junction, , Neuroscience letters, 1984, 10.1016/0304-3940(84)90559-7
Inflammatory damage following first-generation replication-defective adenovirus controlled by anti-LFA-1, , Journal of leukocyte biology, 1997, 10.1002/jlb.61.4.533
Increase in the proliferative capacity of human myoblasts by using the T antigen under the vimentin promoter control, , Muscle & nerve, 1997, 10.1002/(sici)1097-4598(199704)20:43.0.co;2-b
Non-uniform release at the frog neuromuscular junction: evidence of morphological and physiological plasticity, , Brain research, 1987, 10.1016/0165-0173(87)90019-1
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
The paracrine effect: pivotal mechanism in cell-based cardiac repair, , Journal of cardiovascular translational research, 2010, 10.1007/s12265-010-9198-2
Repeated stimulation of the dystrophic mouse neuromuscular junctions, , Muscle & nerve, 1987, 10.1002/mus.880100405
Immunosuppression with FK 506 insures good success of myoblast transplantation in MDX mice, , Transplantation proceedings, 1994
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
Myoblast transplantation: techniques in nonhuman primates as a bridge to clinical trials, , 2011
Myoblast allotransplantation in primates, , Muscle & nerve, 1995
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
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
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
Limb–Girdle Muscular Dystrophies Classification and Therapies, Jacques P. Tremblay, Camille Bouchard, Journal of Clinical Medicine, 2023, 10.3390/jcm12144769
Administration of a soluble activin type IIB receptor promotes the transplantation of human myoblasts in dystrophic mice, , Cell transplantation, 2012, 10.3727/096368911X627480
Dynamics of the early immune cellular reactions after myogenic cell transplantation, , Cell transplantation, 2002, 10.3727/000000002783985378
Expression of dog microdystrophin in mouse and dog muscles by gene therapy, , Molecular therapy : the journal of the American Society of Gene Therapy, 2010, 10.1038/mt.2010.23
Human myoblast transplantation: preliminary results of 4 cases, , Muscle & nerve, 1992, 10.1002/mus.880150504
Myoblast transplantation in non-dystrophic dog, , Neuromuscular disorders : NMD, 1998, 10.1016/s0960-8966(97)00148-x
Scale-up of a myoblast culture process, , Journal of biotechnology, 2001, 10.1016/s0168-1656(01)00291-7
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
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
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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
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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
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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
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Identification of a putative pathway for the muscle homing of stem cells in a muscular dystrophy model, , The Journal of cell biology, 2003, 10.1083/jcb.200210006
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
Use of repeating dispensers to increase the efficiency of the intramuscular myogenic cell injection procedure, , Cell transplantation, 2006, 10.3727/000000006783981648
Telomerase can extend the proliferative capacity of human myoblasts, but does not lead to their immortalization, , Molecular cancer research : MCR, 2003
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Utilization of myoblasts from transgenic mice to evaluate the efficacy of myoblast transplantation, , Muscle & nerve, 1994, 10.1002/mus.880170903
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
Thérapie cellulaire et génique, , 2015
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Dystrophin expression following the transplantation of normal muscle precursor cells protects mdx muscle from contraction-induced damage, , Cell transplantation, 2010, 10.3727/096368910X4863235
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Confirmation of donor-derived dystrophin in a duchenne muscular dystrophy patient allotransplanted with normal myoblasts, , Muscle & nerve, 2016, 10.1002/mus.25129
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
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
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-SCReT (CRISPR-Stop Codon Read Through) method to control Cas9 expression for gene editing, , Gene therapy, 2022, 10.1038/s41434-021-00297-z
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
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
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
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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
First study of intra-arterial delivery of myogenic mononuclear cells to skeletal muscles in primates, , Cell transplantation, 2014, 10.3727/096368914X685032
Male-specific competitive PCR for the quantification of transplanted cells in mice, , BioTechniques, 1999, 10.2144/99273bm05
Mosaic expression of dystrophin in the cerebellum of heterozygote dystrophic (mdx) mice, , Neuromuscular disorders : NMD, 1992, 10.1016/s0960-8966(06)80002-7
Complement deposition and cell death after myoblast transplantation, , Cell transplantation, 1998, 10.1016/s0963-6897(98)00029-3
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
Targeted Gene Addition of Microdystrophin in Mice Skeletal Muscle via Human Myoblast Transplantation, , Molecular therapy. Nucleic acids, 2013, 10.1038/mtna.2012.55
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Myoblast Transplantation in skeletal muscles, , 2010
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CRISPR-induced deletion with SaCas9 restores dystrophin expression in dystrophic models in vitro and in vivo, , 2018, 10.1101/378331
Anti-inflammatory effect of transforming growth factor-beta1 in myoblast transplantation, , Transplantation, 1998, 10.1097/00007890-199803270-00005
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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
Autologous transplantation of muscle precursor cells modified with a lentivirus for muscular dystrophy: human cells and primate models, , Molecular therapy : the journal of the American Society of Gene Therapy, 2007, 10.1038/sj.mt.6300047
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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
Cyclophosphamide immunosuppression does not permit successful myoblast allotransplantation in mouse, , Neuromuscular disorders : NMD, 1995, 10.1016/0960-8966(95)00011-b
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
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
Transplantation of Myogenic Cells in Duchenne Muscular Dystrophy Patients: Clinical Findings, , 2014, 10.1016/b978-0-12-410396-2.00026-8
Implantation of myogenic cells in skeletal muscles, , 2010
Delivery of RNAs to Specific Organs by Lipid Nanoparticles for Gene Therapy, Jacques P. Tremblay, Kelly Godbout, Pharmaceutics, 2022, 10.3390/pharmaceutics14102129
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
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
Cell therapies for inherited myopathies, , Current opinion in rheumatology, 2003, 10.1097/00002281-200311000-00007
Macrophages improve survival, proliferation and migration of engrafted myogenic precursor cells into MDX skeletal muscle, , PloS one, 2012, 10.1371/journal.pone.0046698
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
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
Normal myoblast implantation in MDX mice prevents muscle damage by exercise, , Biochemical and biophysical research communications, 1998, 10.1006/bbrc.1998.9276
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
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
Transcription activator-like effector proteins induce the expression of the frataxin gene, , Human gene therapy, 2012, 10.1089/hum.2012.034
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
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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
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
Myoblast survival enhancement and transplantation success improvement by heat-shock treatment in mdx mice, , Transplantation, 2004, 10.1097/01.tp.0000121503.01535.f5
Labelling of an intermediate saccule of the Golgi apparatus and of parts of the endoplasmic reticulum by a lectin (soybean agglutinin) in the chick ciliary ganglion, , Neuroscience letters, 1983, 10.1016/0304-3940(83)90538-4
Quantification of normal dystrophin mRNA following myoblast transplantation in mdx mice, , Muscle & nerve, 1995, 10.1002/mus.880180909
Boutons originating from the same axon do not participate equally in synaptic transmission, , Neuroscience letters, 1982, 10.1016/0304-3940(82)90342-1
Efficacy and site-specificity of adenoviral vector integration mediated by the phage φC31 integrase, , Human gene therapy methods, 2012, 10.1089/hgtb.2012.122
Clarifying misconceptions about myoblast transplantation in myology, , Molecular therapy : the journal of the American Society of Gene Therapy, 2014, 10.1038/mt.2014.57
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
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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
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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
Human myoblast transplantation: a simple assay for tumorigenicity, , Neuromuscular disorders : NMD, 1991, 10.1016/0960-8966(91)90120-h
Myoblast transplantations lead to the expression of the laminin alpha 2 chain in normal and dystrophic (dy/dy) mouse muscles, , Gene therapy, 1999, 10.1038/sj.gt.3300889
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
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Myoblast transplantation between monozygotic twin girl carriers of Duchenne muscular dystrophy, , Neuromuscular disorders : NMD, 1993, 10.1016/0960-8966(93)90121-y
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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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Pretreatment of myoblast cultures with basic fibroblast growth factor increases the efficacy of their transplantation in mdx mice, , Muscle & nerve, 1995, 10.1002/mus.880180806
Meeting of the Cell Transplantation Society in Miami, , Cell transplantation, 1997
Human myoblast transplantation between immunohistocompatible donors and recipients produces immune reactions, , Transplantation proceedings, 1992
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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
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
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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
Utilization of an antibody specific for human dystrophin to follow myoblast transplantation in nude mice, , Cell transplantation, 1993, 10.1177/096368979300200204
Transplantation of normal and DMD myoblasts expressing the telomerase gene in SCID mice, , Biochemical and biophysical research communications, 2000, 10.1006/bbrc.2000.2735
Gene Therapy in Duchenne’s Muscular Dystrophy, , 2009
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
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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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Depuis 2020/07

Félix Veillette - Maîtrise avec mémoire - En cours
Seyed Mohammadreza Mirinezhad - Post-doctorat - En cours
Ayesha Siddika - Doctorat - En cours
Maheswaran Kesavan - Doctorat - En cours
Gabriel Lamothe - Doctorat - En cours
Camille Bouchard - Doctorat - En cours
Fatima El Husseiny - Doctorat - En cours
Jing Jiang - Doctorat - En cours
Gege Zhao - Doctorat - En cours
Sina Ramezani - Doctorat - En cours
Yaoyao Lu - Doctorat - En cours
Antoine Guyon - Doctorat - 2021/01
Malek Aloui - Maîtrise avec mémoire - 2021/09
Solange Gni-Fiene Yanyabe - Maîtrise avec mémoire - 2022/01
Annabelle Fortin-Archambault - Maîtrise avec mémoire - 2022/05
Guillaume Tremblay - Maîtrise avec mémoire - 2022/05
Pouiré Yameogo - Doctorat - 2023/01
Cedric Happi Mbakam - Doctorat - 2023/05
Kelly Godbout - Maîtrise avec mémoire - 2024/01
Bo Song - Maîtrise avec mémoire - 2024/01

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