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ArticlesPeer ReviewedFull Text
Delivery of RNAs to Specific Organs by Lipid Nanoparticles for Gene Therapy
Author / Creator:Godbout, Kelly ; Tremblay, Jacques P.
Publisher:Basel: MDPI AG
Journal title:Pharmaceutics, 2022-10, Vol.14 (10), p.2129
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ArticlesPeer ReviewedFull Text
Prime Editing for Human Gene Therapy: Where Are We Now?
Author / Creator:Godbout, Kelly ; Tremblay, Jacques P.
Publisher:Switzerland: MDPI AG
Journal title:Cells (Basel, Switzerland), 2023-02, Vol.12 (4), p.536
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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
Author / Creator:Guyon, Antoine ; Rousseau, Joël ; Lamothe, Gabriel ; Tremblay, Jacques P.
Publisher:United States: Public Library of Science
Journal title:PloS one, 2020-12, Vol.15 (12), p.e0237122-e0237122
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ArticlesPeer ReviewedFull Text
Small-molecule inhibitors of proteasome increase CjCas9 protein stability
Author / Creator:Yaméogo, Pouiré ; Majeau, Nathalie ; Mbakam, Cedric Happi ; Tremblay, Jacques P.
Publisher:United States: Public Library of Science
Journal title:PloS one, 2023-01, Vol.18 (1), p.e0280353-e0280353
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ArticlesPeer ReviewedFull Text
CRISPR-SCReT (CRISPR-Stop Codon Read Through) method to control Cas9 expression for gene editing
Author / Creator:Yaméogo, Pouiré ; Duchêne, Benjamin L. ; Majeau, Nathalie ; Tremblay, Jacques P.
Publisher:London: Nature Publishing Group UK
Journal title:Gene therapy, 2022-04, Vol.29 (3-4), p.171-177
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Reading Frame Correction by Targeted Genome Editing Restores Dystrophin Expression in Cells From Duchenne Muscular Dystrophy Patients
Author / Creator:Ousterout, David G ; Perez-Pinera, Pablo ; Thakore, Pratiksha I ; Kabadi, Ami M ; Brown, Matthew T ; Qin, Xiaoxia ; Fedrigo, Olivier ; Mouly, Vincent ; Tremblay, Jacques P ; Gersbach, Charles A
Publisher:United States: Elsevier Inc
Journal title:Molecular therapy, 2013-09, Vol.21 (9), p.1718-1726
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Successful Correction by Prime Editing of a Mutation in the RYR1 Gene Responsible for a Myopathy
Author / Creator:Godbout, Kelly ; Rousseau, Joël ; Tremblay, Jacques P.
Publisher:Switzerland: MDPI AG
Journal title:Cells (Basel, Switzerland), 2023-12, Vol.13 (1), p.31
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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?
Author / Creator:Skuk, Daniel ; Tremblay, Jacques P
Publisher:Los Angeles, CA: SAGE Publications
Journal title:Cell transplantation, 2020-01, Vol.29, p.96368972093912-963689720939120
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Clarifying Misconceptions About Myoblast Transplantation in Myology
Author / Creator:Skuk, Daniel ; Tremblay, Jacques P
Publisher:United States: Elsevier Inc
Journal title:Molecular therapy, 2014-05, Vol.22 (5), p.897-898
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Current Status of Pharmaceutical and Genetic Therapeutic Approaches to Treat DMD
Author / Creator:Pichavant, Christophe ; Aartsma-Rus, Annemieke ; Clemens, Paula R ; Davies, Kay E ; Dickson, George ; Takeda, Shin'ichi ; Wilton, Steve D ; Wolff, Jon A ; Wooddell, Christine I ; Xiao, Xiao ; Tremblay, Jacques P
Publisher:United States: Elsevier Inc
Journal title:Molecular therapy, 2011-05, Vol.19 (5), p.830-840
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ArticlesPeer ReviewedFull Text
Emerging Perspectives on Prime Editor Delivery to the Brain
Author / Creator:BenDavid, Eli ; Ramezanian, Sina ; Lu, Yaoyao ; Rousseau, Joël ; Schroeder, Avi ; Lavertu, Marc ; Tremblay, Jacques P.
Publisher:Switzerland: MDPI AG
Journal title:Pharmaceuticals (Basel, Switzerland), 2024-06, Vol.17 (6), p.763
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ArticlesPeer ReviewedFull Text
Increased Expression of Laminin Subunit Alpha 1 Chain by dCas9-VP160
Author / Creator:Perrin, Arnaud ; Rousseau, Joël ; Tremblay, Jacques P.
Publisher:United States: Elsevier Inc
Journal title:Molecular therapy. Nucleic acids, 2017-03, Vol.6, p.68-79
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ArticlesPeer ReviewedFull Text
Efficient Restoration of the Dystrophin Gene Reading Frame and Protein Structure in DMD Myoblasts Using the CinDel Method
Author / Creator:Iyombe-Engembe, Jean-Paul ; Ouellet, Dominique L ; Barbeau, Xavier ; Rousseau, Joël ; Chapdelaine, Pierre ; Lagüe, Patrick ; Tremblay, Jacques P
Publisher:United States: Elsevier Inc
Journal title:Molecular therapy. Nucleic acids, 2016, Vol.5 (1), p.e283-e283, Article e283
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ArticlesPeer ReviewedFull Text
Cell Therapy in Myology: Dynamics of Muscle Precursor Cell Death after Intramuscular Administration in Non-human Primates
Author / Creator:Skuk, Daniel ; Tremblay, Jacques P.
Publisher:United States: Elsevier Limited
Journal title:Molecular therapy. Methods & clinical development, 2017-06, Vol.5 (C), p.232-240
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ArticlesPeer ReviewedFull Text
Macrophages Improve Survival, Proliferation and Migration of Engrafted Myogenic Precursor Cells into MDX Skeletal Muscle
Author / Creator:Lesault, Pierre-François ; Theret, Marine ; Magnan, Mélanie ; Cuvellier, Sylvain ; Niu, Yiming ; Gherardi, Romain K. ; Tremblay, Jacques P. ; Hittinger, Luc ; Chazaud, Bénédicte
Publisher:United States: Public Library of Science
Journal title:PloS one, 2012-10, Vol.7 (10), p.e46698-e46698
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ArticlesPeer ReviewedFull Text
Increased Frataxin Expression Induced in Friedreich Ataxia Cells by Platinum TALE-VP64s or Platinum TALE-SunTag
Author / Creator:Cherif, Khadija ; Gérard, Catherine ; Rousseau, Joël ; Ouellet, Dominique L. ; Chapdelaine, Pierre ; Tremblay, Jacques P.
Publisher:United States: Elsevier Inc
Journal title:Molecular therapy. Nucleic acids, 2018-09, Vol.12, p.19-32
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Myogenic progenitor cells derived from human induced pluripotent stem cell are immune‐tolerated in humanized mice
Author / Creator:Benabdallah, Basma ; Désaulniers‐Langevin, Cynthia ; Goyer, Marie‐Lyn ; Colas, Chloé ; Maltais, Chantale ; Li, Yuanyi ; Guimond, Jean V. ; Tremblay, Jacques P. ; Haddad, Elie ; Beauséjour, Christian
Publisher:Hoboken, USA: John Wiley & Sons, Inc
Journal title:Stem cells translational medicine, 2021-02, Vol.10 (2), p.267-277
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ArticlesPeer ReviewedFull Text
Dystrophin Expression in Muscles of Duchenne Muscular Dystrophy Patients After High-Density Injections of Normal Myogenic Cells
Author / Creator:Skuk, Daniel ; Goulet, Marlyne ; Roy, Brigitte ; Chapdelaine, Pierre ; Bouchard, Jean-Pierre ; Roy, Raynald ; Dugré, Francine J. ; Sylvain, Michel ; Lachance, Jean-Guy ; Deschênes, Louise ; Senay, Hélène ; Tremblay, Jacques P.
Publisher:Hagerstown, MD: American Association of Neuropathologists, Inc
Journal title:Journal of neuropathology and experimental neurology, 2006-04, Vol.65 (4), p.371-386
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Intramuscular Transplantation of Human Postnatal Myoblasts Generates Functional Donor-Derived Satellite Cells
Author / Creator:Skuk, Daniel ; Paradis, Martin ; Goulet, Marlyne ; Chapdelaine, Pierre ; Rothstein, David M ; Tremblay, Jacques P
Publisher:United States: Elsevier Inc
Journal title:Molecular therapy, 2010-09, Vol.18 (9), p.1689-1697
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ArticlesPeer ReviewedFull Text
CRISPR-Cas9 Gene Therapy for Duchenne Muscular Dystrophy
Author / Creator:Happi Mbakam, Cedric ; Lamothe, Gabriel ; Tremblay, Guillaume ; Tremblay, Jacques P.
Publisher:Cham: Springer International Publishing
Journal title:Neurotherapeutics, 2022-04, Vol.19 (3), p.931-941
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Delivery of RNAs to Specific Organs by Lipid Nanoparticles for Gene Therapy

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Prime Editing for Human Gene Therapy: Where Are We Now?

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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

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Small-molecule inhibitors of proteasome increase CjCas9 protein stability

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CRISPR-SCReT (CRISPR-Stop Codon Read Through) method to control Cas9 expression for gene editing

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Reading Frame Correction by Targeted Genome Editing Restores Dystrophin Expression in Cells From Duchenne Muscular Dystrophy Patients

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Successful Correction by Prime Editing of a Mutation in the RYR1 Gene Responsible for a Myopathy

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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?

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Clarifying Misconceptions About Myoblast Transplantation in Myology

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Current Status of Pharmaceutical and Genetic Therapeutic Approaches to Treat DMD

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Emerging Perspectives on Prime Editor Delivery to the Brain

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Increased Expression of Laminin Subunit Alpha 1 Chain by dCas9-VP160

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Efficient Restoration of the Dystrophin Gene Reading Frame and Protein Structure in DMD Myoblasts Using the CinDel Method

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Cell Therapy in Myology: Dynamics of Muscle Precursor Cell Death after Intramuscular Administration in Non-human Primates

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Macrophages Improve Survival, Proliferation and Migration of Engrafted Myogenic Precursor Cells into MDX Skeletal Muscle

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Increased Frataxin Expression Induced in Friedreich Ataxia Cells by Platinum TALE-VP64s or Platinum TALE-SunTag

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Myogenic progenitor cells derived from human induced pluripotent stem cell are immune‐tolerated in humanized mice

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Dystrophin Expression in Muscles of Duchenne Muscular Dystrophy Patients After High-Density Injections of Normal Myogenic Cells

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Intramuscular Transplantation of Human Postnatal Myoblasts Generates Functional Donor-Derived Satellite Cells

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CRISPR-Cas9 Gene Therapy for Duchenne Muscular Dystrophy

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