Delivery of RNAs to Specific Organs by Lipid Nanoparticles for Gene Therapy von Godbout, Kelly, Tremblay, Jacques P

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The protective mutation A673T in amyloid precursor protein gene decreases A beta peptides production for 14 forms of Familial Alzheimer's Disease in SH-SY5Y cells von Guyon, Antoine, Rousseau, Joel, Lamothe, Gabriel, Tremblay, Jacques P.

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Prime Editing Permits the Introduction of Specific Mutations in the Gene Responsible for Duchenne Muscular Dystrophy von Happi Mbakam, Cédric, Rousseau, Joël, Tremblay, Guillaume, Yameogo, Pouiré, Tremblay, Jacques P

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Prime Editing for Human Gene Therapy: Where Are We Now? von Godbout, Kelly, Tremblay, Jacques P

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Small-molecule inhibitors of proteasome increase CjCas9 protein stability von Yaméogo, Pouiré, Majeau, Nathalie, Mbakam, Cedric Happi, Tremblay, Jacques P

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CRISPR-Cas9 delivery strategies with engineered extracellular vesicles von Lu, Yaoyao, Godbout, Kelly, Lamothe, Gabriel, Tremblay, Jacques P.

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CRISPR-SCReT (CRISPR-Stop Codon Read Through) method to control Cas9 expression for gene editing von Yaméogo, Pouiré, Duchêne, Benjamin L., Majeau, Nathalie, Tremblay, Jacques P.

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

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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 Quo... von Skuk, Daniel, Tremblay, Jacques P

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Therapeutic Strategies for Dystrophin Replacement in Duchenne Muscular Dystrophy von Happi Mbakam, Cedric, Lamothe, Gabriel, Tremblay, Jacques P

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Successful Correction by Prime Editing of a Mutation in the RYR1 Gene Responsible for a Myopathy von Godbout, Kelly, Rousseau, Joël, Tremblay, Jacques P

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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 von Hui, Chin Wai, St-Pierre, Marie-Kim, Detuncq, Jérôme, Aumailley, Lucie, Dubois, Marie-Julie, Couture, Vanessa, Skuk, Daniel, Marette, André, Tremblay, Jacques P., Lebel, Michel, Tremblay, Marie-Ève

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Clarifying Misconceptions About Myoblast Transplantation in Myology von Skuk, Daniel, Tremblay, Jacques P

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Prime editing optimized RTT permits the correction of the c.8713C>T mutation in DMD gene von Happi Mbakam, Cedric, Rousseau, Joel, Lu, Yaoyao, Bigot, Anne, Mamchaoui, Kamel, Mouly, Vincent, Tremblay, Jacques P.

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Current Status of Pharmaceutical and Genetic Therapeutic Approaches to Treat DMD von 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

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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 von Guyon, Antoine, Rousseau, Joël, Bégin, Francis-Gabriel, Bertin, Tom, Lamothe, Gabriel, Tremblay, Jacques P.

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Emerging Perspectives on Prime Editor Delivery to the Brain von BenDavid, Eli, Ramezanian, Sina, Lu, Yaoyao, Rousseau, Joël, Schroeder, Avi, Lavertu, Marc, Tremblay, Jacques P

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Improvements of nuclease and nickase gene modification techniques for the treatment of genetic diseases von Lu, Yaoyao, Happi Mbakam, Cedric, Song, Bo, Bendavid, Eli, Tremblay, Jacques-P.

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Increased Expression of Laminin Subunit Alpha 1 Chain by dCas9-VP160 von Perrin, Arnaud, Rousseau, Joël, Tremblay, Jacques P.

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Intramuscular Transplantation of Myogenic Cells in Primates: Importance of Needle Size, Cell Number, and Injection Volume von Skuk, Daniel, Goulet, Marlyne, Tremblay, Jacques P.

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