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Molecular Therapy. Methods & Clinical Development|August 21, 2023
Adeno-associated virus-mediated gene therapy in a patient with Canavan disease using dual routes of administration and immune modulationManuela Corti, Barry J Byrne, Dominic J Gessler, et al.Plos One|July 27, 2010
Identification of pathway-biased and deleterious melatonin receptor mutants in autism spectrum disorders and in the general populationPauline Chaste, Nathalie Clement, Oriane Mercati, et al.Human Molecular Genetics|April 27, 2011
Pompe disease gene therapyBarry J Byrne, Darin J Falk, Christina A Pacak, et al.Human Gene Therapy|February 19, 2010
Adeno-associated virus-mediated correction of a canine model of glycogen storage disease type IaDavid A Weinstein, Catherine E Correia, Thomas Conlon, et al.Journal of Pineal Research|May 28, 2011
Genetic variations of the melatonin pathway in patients with attention-deficit and hyperactivity disordersPauline Chaste, Nathalie Clement, Hany Goubran Botros, et al.International Journal of Cancer|April 25, 2019
Study of chromatin remodeling genes implicates SMARCA4 as a putative player in oncogenesis in neuroblastomaAngela Bellini, Nadia Bessoltane-Bentahar, Jaydutt Bhalshankar, et al.Clinical Cancer Research : an Official Journal of the American Association for Cancer Research|July 22, 2016
Genomic Copy Number Profiling Using Circulating Free Tumor DNA Highlights Heterogeneity in NeuroblastomaMathieu Chicard, Sandrine Boyault, Leo Colmet Daage, et al.Science Translational Medicine|January 4, 2023
Assessment of systemic AAV-microdystrophin gene therapy in the GRMD model of Duchenne muscular dystrophySharla M Birch, Michael W Lawlor, Thomas J Conlon, et al.Pageof 3