Jacques-P. Tremblay

• Centre de recherche du CHU de Québec - Université Laval
• Centre thématique de recherche en neurosciences

Financements des 2 dernières années

• Ayesha Siddika, Doctorat en médecine moléculaire
• Fatima El Husseiny, Doctorat en médecine moléculaire
• Félix Veillette, Doctorat en médecine moléculaire
• Félix Veillette, Maîtrise en médecine moléculaire - avec mémoire
• Frank Simard, Maîtrise en médecine moléculaire - avec mémoire
• Gege Zhao, Doctorat en médecine moléculaire
• Jing Jiang, Doctorat en médecine moléculaire
• Maheswaran Kesavan, Doctorat en médecine moléculaire
• Sina Ramezani, Doctorat en médecine moléculaire

Encadrements des 5 dernières années

• Annabelle Fortin-Archambault, Maîtrise en médecine moléculaire - avec mémoire
• Bo Song, Maîtrise en médecine moléculaire - avec mémoire
• Camille Bouchard, Maîtrise en médecine moléculaire - avec mémoire
• Camille Bouchard, Doctorat en médecine moléculaire
• Cedric Happi Mbakam, Doctorat en médecine moléculaire
• Gabriel Lamothe, Doctorat en médecine moléculaire
• Gabriel Lamothe, Maîtrise en médecine moléculaire - avec mémoire
• Guillaume Tremblay, Maîtrise en sciences cliniques et biomédicales - avec mémoire
• Kelly Godbout, Maîtrise en médecine moléculaire - avec mémoire
• Pouiré Yameogo, Doctorat en médecine moléculaire
• Seyed Mohammadreza Mirinezhad, Stage postdoctoral en médecine
• Solange Gni-Fiene Yanyabe, Maîtrise en médecine moléculaire - avec mémoire
• Yaoyao Lu, Doctorat en médecine moléculaire

Travaux de recherche des 5 dernières années

Prime Editing Mediated Generation and Correction of the mdx5cv Mutation Restores Dystrophin Expression in Myoblasts International Journal of Molecular Sciences, 2026. Ayesha Siddika, Fatima El Husseiny, Joël Rousseau, Jacques P. Tremblay. DOI 10.3390/ijms27156927

Gene Editing Strategies for Duchenne Muscular Dystrophy: From Molecular Mechanisms to Clinical Translation Cells, 2026. Ayesha Siddika, Joël Rousseau, Félix Veillette, Camille Bouchard, Yaoyao Lu, Jacques P. Tremblay. DOI 10.3390/cells15100852

Molecular characterization of a new R1925X point mutation mouse model for dysferlinopathy Genes & Diseases, 2026. Camille Bouchard, Louis Despax, Joël Rousseau, Jacques P. Tremblay. DOI 10.1016/j.gendis.2025.101885

Successful In Vitro Modification of the Dmd Gene Using Prime Editing Cells, 2026. Ayesha Siddika, Fatima El Husseiny, Joël Rousseau, Jacques P. Tremblay. DOI 10.3390/cells15090740

The Improvements and Applications of Prime Editing DNA, 2026. Yaoyao Lu, Camille Bouchard, Nicolas Soucy, Ayesha Siddika, Gabriel Lamothe, Kelly Godbout, Jacques P. Tremblay. DOI 10.3390/dna6010016

In Vitro Correction of Point Mutations in the DYSF Gene Using Prime Editing International Journal of Molecular Sciences, 2025. Camille Bouchard, Joël Rousseau, Gabriel Lamothe, Marie Dubost, Laura Bastrenta, Sina Ramezani, Jacques P. Tremblay. DOI 10.3390/ijms26125647

Universal Prime Editing Therapeutic Strategy for RyR1-Related Myopathies: A Protective Mutation Rescues Leaky RyR1 Channel International Journal of Molecular Sciences, 2025. Kelly Godbout, Mathieu Dugas, Steven R. Reiken, Sina Ramezani, Alexia Falle, Joël Rousseau, Anetta E. Wronska, Gabriel Lamothe, Geoffrey Canet, Yaoyao Lu, Emmanuel Planel, Andrew R. Marks, Jacques P. Tremblay. DOI 10.3390/ijms26072835

Emerging Perspectives on Prime Editor Delivery to the Brain Pharmaceuticals, 2024. Eli BenDavid, Sina Ramezanian, Yaoyao Lu, Joël Rousseau, Avi Schroeder, Marc Lavertu, Jacques P. Tremblay. DOI 10.3390/ph17060763

Successful Correction by Prime Editing of a Mutation in the RYR1 Gene Responsible for a Myopathy Cells, 2023. Kelly Godbout, Joël Rousseau, Jacques P. Tremblay. DOI 10.3390/cells13010031

Portrait of Dysferlinopathy: Diagnosis and Development of Therapy Journal of Clinical Medicine, 2023. Camille Bouchard, Jacques P. Tremblay. DOI 10.3390/jcm12186011

CRISPR/Cas9 delivery strategies with engineered extracellular vesicles Molecular Therapy - Nucleic Acids, 2023. Yaoyao Lu, Kelly Godbout, Gabriel Lamothe, Jacques P. Tremblay. DOI 10.1016/j.omtn.2023.102040

Finding an Appropriate Mouse Model to Study the Impact of a Treatment for Friedreich Ataxia on the Behavioral Phenotype Genes, 2023. Camille Bouchard, Catherine Gérard, Solange Gni-fiene Yanyabé, Nathalie Majeau, Malek Aloui, Gabrielle Buisson, Pouiré Yameogo, Vanessa Couture, Jacques P. Tremblay. DOI 10.3390/genes14081654

Removal of the GAA repeat in the heart of a Friedreich’s ataxia mouse model using CjCas9 Gene Therapy, 2023. Pouiré Yaméogo, Catherine Gérard, Nathalie Majeau, Jacques P. Tremblay. DOI 10.1038/s41434-023-00387-0

Limb–Girdle Muscular Dystrophies Classification and Therapies Journal of Clinical Medicine, 2023. Camille Bouchard, Jacques P. Tremblay. DOI 10.3390/jcm12144769

Prime Editing for Human Gene Therapy: Where Are We Now? Cells, 2023. Kelly Godbout, Jacques P. Tremblay. DOI 10.3390/cells12040536

Small-molecule inhibitors of proteasome increase CjCas9 protein stability PLOS ONE, 2023. Pouiré Yaméogo, Nathalie Majeau, Cedric Happi Mbakam, Jacques P. Tremblay. DOI 10.1371/journal.pone.0280353

Prime editing optimized RTT permits the correction of the c.8713C>T mutation in DMD gene Molecular Therapy - Nucleic Acids, 2022. Cedric Happi Mbakam, Joel Rousseau, Yaoyao Lu, Anne Bigot, Kamel Mamchaoui, Vincent Mouly, Jacques P. Tremblay. DOI 10.1016/j.omtn.2022.09.022

Delivery of RNAs to Specific Organs by Lipid Nanoparticles for Gene Therapy Pharmaceutics, 2022. Kelly Godbout, Jacques P. Tremblay. DOI 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. DOI 10.1016/j.ymthe.2022.05.023

CRISPR-SCReT (CRISPR-Stop Codon Read Through) method to control Cas9 expression for gene editing Gene therapy, 2022. DOI 10.1038/s41434-021-00297-z

Therapeutic Strategies for Dystrophin Replacement in Duchenne Muscular Dystrophy Frontiers in medicine, 2022. DOI 10.3389/fmed.2022.859930

CRISPR-Cas9 Gene Therapy for Duchenne Muscular Dystrophy Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics, 2022. DOI 10.1007/s13311-022-01197-9

Insertion of the Icelandic Mutation (A673T) by Prime Editing: A Potential Preventive Treatment for Familial and Sporadic Alzheimer's Disease The CRISPR journal, 2022. DOI 10.1089/crispr.2021.0085

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. DOI 10.1016/j.omtn.2021.02.032

Three Decades of Clinical Gene Therapy: From Experimental Technologies to Viable Treatments Molecular therapy : the journal of the American Society of Gene Therapy, 2021. DOI 10.1016/j.ymthe.2021.01.013

Myogenic progenitor cells derived from human induced pluripotent stem cell are immune-tolerated in humanized mice Stem cells translational medicine, 2021. DOI 10.1002/sctm.19-0452

Current Clinical Applications of In Vivo Gene Therapy with AAVs Molecular therapy : the journal of the American Society of Gene Therapy, 2021. DOI 10.1016/j.ymthe.2020.12.007