User profiles for Caroline Le Guiner
 | Le Guiner Caroline, PhDTaRGeT lab (Translational Research in Gene Therapy), INSERM UMR 1089, Nantes … Verified email at univ-nantes.fr Cited by 2806 |
Adeno-associated virus vector genomes persist as episomal chromatin in primate muscle
M Penaud-Budloo, C Le Guiner, A Nowrouzi… - Journal of …, 2008 - Am Soc Microbiol
Recombinant adeno-associated virus (rAAV) vectors are capable of mediating long-term
gene expression following administration to skeletal muscle. In rodent muscle, the vector …
gene expression following administration to skeletal muscle. In rodent muscle, the vector …
[HTML][HTML] Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy
Duchenne muscular dystrophy (DMD) is an incurable X-linked muscle-wasting disease
caused by mutations in the dystrophin gene. Gene therapy using highly functional …
caused by mutations in the dystrophin gene. Gene therapy using highly functional …
The RNA-binding protein TIA-1 is a novel mammalian splicing regulator acting through intron sequences adjacent to a 5′ splice site
…, CF Bourgeois, C Le Guiner… - … and cellular biology, 2000 - Taylor & Francis
Splicing of the K-SAM alternative exon of the fibroblast growth factor receptor 2 gene is
heavily dependent on the U-rich sequence IAS1 lying immediately downstream from its 5′ …
heavily dependent on the U-rich sequence IAS1 lying immediately downstream from its 5′ …
[HTML][HTML] Characterization of dystrophin deficient rats: a new model for Duchenne muscular dystrophy
…, S Remy, V Thepenier, V François, C Le Guiner… - PloS one, 2014 - journals.plos.org
A few animal models of Duchenne muscular dystrophy (DMD) are available, large ones such
as pigs or dogs being expensive and difficult to handle. Mdx (X-linked muscular dystrophy) …
as pigs or dogs being expensive and difficult to handle. Mdx (X-linked muscular dystrophy) …
In vivo gene regulation using tetracycline-regulatable systems
K Stieger, B Belbellaa, C Le Guiner, P Moullier… - Advanced drug delivery …, 2009 - Elsevier
Numerous preclinical studies have demonstrated the efficacy of viral gene delivery vectors,
and recent clinical trials have shown promising results. However, the tight control of …
and recent clinical trials have shown promising results. However, the tight control of …
[HTML][HTML] Forelimb treatment in a large cohort of dystrophic dogs supports delivery of a recombinant AAV for exon skipping in Duchenne patients
C Le Guiner, M Montus, L Servais, Y Cherel… - Molecular Therapy, 2014 - cell.com
Duchenne muscular dystrophy (DMD) is a severe muscle-wasting disorder caused by mutations
in the dystrophin gene, without curative treatment yet available. Our study provides, for …
in the dystrophin gene, without curative treatment yet available. Our study provides, for …
[HTML][HTML] TIA-1 and TIAR activate splicing of alternative exons with weak 5′ splice sites followed by a U-rich stretch on their own pre-mRNAs
C Le Guiner, F Lejeune, D Galiana, L Kister… - Journal of Biological …, 2001 - ASBMB
TIA-1 has recently been shown to activate splicing of specific pre-mRNAs transcribed from
transiently transfected minigenes, and of some 5′ splice sites in vitro, but has not been …
transiently transfected minigenes, and of some 5′ splice sites in vitro, but has not been …
[HTML][HTML] Systemic delivery of allogenic muscle stem cells induces long-term muscle repair and clinical efficacy in duchenne muscular dystrophy dogs
Duchenne muscular dystrophy (DMD) is a genetic progressive muscle disease resulting
from the lack of dystrophin and without effective treatment. Adult stem cell populations have …
from the lack of dystrophin and without effective treatment. Adult stem cell populations have …
[HTML][HTML] Serum profiling identifies novel muscle miRNA and cardiomyopathy-related miRNA biomarkers in Golden Retriever muscular dystrophy dogs and Duchenne …
…, S Krimi, J Buisset, F Amor, C Le Guiner… - The American journal of …, 2014 - Elsevier
Duchenne muscular dystrophy (DMD) is a fatal, X-linked neuromuscular disease that affects
1 boy in 3500 to 5000 boys. The golden retriever muscular dystrophy dog is the best …
1 boy in 3500 to 5000 boys. The golden retriever muscular dystrophy dog is the best …
[HTML][HTML] Integration frequency and intermolecular recombination of rAAV vectors in non-human primate skeletal muscle and liver
…, M Penaud-Budloo, C Kaeppel, U Appelt, C Le Guiner… - Molecular Therapy, 2012 - cell.com
The comprehensive characterization of recombinant adeno-associated viral (rAAV)
integration frequency and persistence for assessing rAAV vector biosafety in gene therapy is …
integration frequency and persistence for assessing rAAV vector biosafety in gene therapy is …