Forelimb Treatment in a Large Cohort of Dystrophic Dogs Supports Delivery of a Recombinant AAV for Exon Skipping in Duchenne Patients
2014 · Molecular Therapy · 114 citations
https://doi.org/10.1038/mt.2014.151
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Caroline Le Guiner
Inserm(FR)
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Caroline Le Guiner is an academic researcher from Inserm. The author has contributed to research in topics: Muscle Physiology and Disorders & Virus-based gene therapy research & RNA Interference and Gene Delivery. The author has an h-index of 26, co-authored 94 publications.
ORCID: 0000-0002-6904-2300Papers by this author
worksLiver-Specific Transcriptional Modules Identified by Genome-Wide In Silico Analysis Enable Efficient Gene Therapy in Mice and Non-Human Primates
2014 · Molecular Therapy · 77 citations
https://doi.org/10.1038/mt.2014.114
Levels of inflammation and oxidative stress, and a role for taurine in dystropathology of the Golden Retriever Muscular Dystrophy dog model for Duchenne Muscular Dystrophy
2016 · Redox Biology · 69 citations
https://doi.org/10.1016/j.redox.2016.08.016
AAV2/9-mediated silencing of PMP22 prevents the development of pathological features in a rat model of Charcot-Marie-Tooth disease 1 A
2021 · Nature Communications · 67 citations
https://doi.org/10.1038/s41467-021-22593-3
Dystrophin and mini-dystrophin quantification by mass spectrometry in skeletal muscle for gene therapy development in Duchenne muscular dystrophy
2021 · Gene Therapy · 29 citations
https://doi.org/10.1038/s41434-021-00300-7
Myostatin Is a Quantifiable Biomarker for Monitoring Pharmaco-gene Therapy in Duchenne Muscular Dystrophy
2020 · Molecular Therapy — Methods & Clinical Development · 26 citations
https://doi.org/10.1016/j.omtm.2020.06.016
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