Caroline Le Guiner

This study shows that intracellular accumulation of AAV6 genomes in microglia drives reactivity and inflammation in retinal gene therapy.

This study uses engineered human muscle tissues to evaluate AAV capsid efficiency, finding that more mature tissues show improved transduction by AAV9 and MyoAAV variants.

This study enhances liver gene therapy by conjugating GalNAc to rAAV capsids, improving transduction efficiency in mice while reducing required dosages.

This study identifies endosomal pathway dysfunction in DMD muscle cells and shows that Rab5 upregulation contributes to endosomal defects, which can be mitigated by Rab5 knock-down or dystrophin restoration.

This study shows that dystrophin deficiency disrupts cell junction formation during embryonic myogenesis in human pluripotent stem cells.

This study shows that AAV2/9-mediated PMP22 silencing in rats prevents CMT1A disease and identifies skin biomarkers as potential outcome measures.