Caroline Le Guiner

Inserm — France

Biochemistry, Genetics and Molecular Biology · Life Sciences

26h-index2.6kcitations109works2.72yr avg
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Research Topics

Muscle Physiology and Disorders(53), Virus-based gene therapy research(52), RNA Interference and Gene Delivery(27), CRISPR and Genetic Engineering(21), Tissue Engineering and Regenerative Medicine(13)

Publications109 total

Engineered muscle tissues with enhanced maturation enable the identification of clinically relevant rAAV capsids
2026Open Access
Dose- and genotype-dependent cardiac arrhythmia and sudden death in rats following microdystrophin gene therapy
Molecular Therapy·2025Open Access
Transcriptomic profiling of skeletal muscle in the DMDmdx rat model of Duchenne muscular dystrophy
Scientific Reports·2025· 1 citedOpen Access
Advancing liver gene therapy: Enhanced transduction with GalNAc-bioconjugated rAAV capsids
Biomedicine & Pharmacotherapy·2025Open Access
Early Endosome Disturbance and Endolysosomal Pathway Dysfunction in Duchenne Muscular Dystrophy
American Journal Of Pathology·2025· 1 citedOpen Access
Early endosome disturbance and endolysosomal pathway dysfunction in Duchenne muscular dystrophy
2024· 1 citedOpen Access
Advancing Liver Gene Therapy: Enhanced Transduction with GalNAc-Bioconjugated rAAV Capsids
2024
Dystrophin deficiency impairs cell junction formation during embryonic myogenesis from pluripotent stem cells
iScience·2024· 4 citedOpen Access
Novel chemical tyrosine functionalization of adeno-associated virus improves gene transfer efficiency in liver and retina
Biomedicine & Pharmacotherapy·2024· 17 citedOpen Access
Dystrophin deficiency impairs cell junction formation during embryonic myogenesis
2023· 2 citedOpen Access
Evaluation of an AAV9-mini-dystrophin gene therapy candidate in a rat model of Duchenne muscular dystrophy
Molecular Therapy — Methods & Clinical Development·2023· 17 citedOpen Access
Characterization of the muscular and cardiac diseases of the DMSXL mouse model, a transgenic mouse model for Myotonic Dystrophy type 1
SPIRE - Sciences Po Institutional REpository·2023Open Access
Novel chemical tyrosine functionalization of adeno-associated virus improves gene transfer efficiency in liver and retina
2023· 4 citedOpen Access
Intravitreal air tamponade after AAV2 subretinal injection modifies retinal EGFP distribution
Molecular Therapy — Methods & Clinical Development·2023· 15 citedOpen Access
Muscle Pathology in Dystrophic Rats and Zebrafish Is Unresponsive to Taurine Treatment, Compared to the mdx Mouse Model for Duchenne Muscular Dystrophy
Metabolites·2023· 3 citedOpen Access
Mannose-coupled AAV2: A second-generation AAV vector for increased retinal gene therapy efficiency
Molecular Therapy — Methods & Clinical Development·2022· 3 citedOpen Access
AAV2/9-mediated gene transfer into murine lacrimal gland leads to a long-term targeted tear film modification
Molecular Therapy — Methods & Clinical Development·2022· 7 citedOpen Access
AAV2/9-mediated gene transfer into murine lacrimal gland leads to a long-term targeted tear film modification
2022Open Access
Evaluation of the dystrophin carboxy-terminal domain for micro-dystrophin gene therapy in cardiac and skeletal muscles in the DMDmdx rat model
Gene Therapy·2022· 17 cited
TRPC3, but not TRPC1, as a good therapeutic target for standalone or complementary treatment of DMD
Journal of Translational Medicine·2021· 15 citedOpen Access
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Frequent Co-authors

Philippe Moullier(25), Oumeya Adjali(15), Thibaut Larcher(14), Yan Chérel(11), Richard O. Snyder(10), Laurent Servais(8), Virginie François(8), Ignacio Anegón(8), Mireille Ledevin(8), Fabienne Rolling(7), Jack‐Yves Deschamps(7), Lydie Guigand(7), Mickaël Guilbaud(7), Magalie Penaud‐Budloo(6), Alice Toromanoff(6), Marie Montus(6), Jean‐Yves Hogrel(6), Thomas Voit(6), Aude Lafoux(6), Corinne Huchet(6)