Genethon (France)

Recent papers from Genethon (France)

1. Human iPSC-derived liver organoid model multicellular tissue responses and therapeutic rescue in Wolman diseaseCell Death and Disease · 2026
2. S -Nitrosylation of glycogen phosphorylase regulates nuclear glycogenolysis and is impaired in metabolic liver disordersScience Advances · 2026
3. Bridging the translational gap in drug repurposing for rare diseases: clinical success versus regulatory conversionDrug Discovery Today · 2026
4. Targeting white matter astrocytes with a Gfap-disruptive Cas9 nuclease benefits a murine model of Alexander diseaseMolecular Therapy · 2026
5. Capsid stabilization reprograms the nuclear fate of the HIV genomeScience Advances · 2026
6. Rapamycin nanoparticles mitigate anti-AAV antibody formation in a mouse model of ornithine transcarbamylase deficiencyMolecular Therapy Advances · 2026
7. Flunarizine changes microRNA expression in cell cultures and in a mouse model of spinal muscular atrophyScientific Reports · 2026
8. CD8 T-cell activation occurs 24 hours post-AAV administration and is driven by muscle promoter specificityJCI Insight · 2026
9. Impact of downstream purification process and AAV serotype on protein-impurity clearanceMolecular Therapy Advances · 2026
10. TREAT-NMD advisory committee for therapeutics: Preclinical and clinical learnings from 15 years of TACTJournal of Neuromuscular Diseases · 2026
11. Author Correction: Disease exacerbation in human DMD MYOrganoids enables gene therapy evaluation and unveils persistence of fibrotic activitynpj Regenerative Medicine · 2026
12. Illuminating “the dark side of the HBB”: Visualizing the full spectrum of Cas9 outcomesMolecular Therapy — Nucleic Acids · 2026
13. 2D Multimodal Image Collection for Fluorescence Prediction from Transmitted Light MicroscopyScientific Data · 2026
14. DAB2 in LGMD R2: a molecular link between disease progression and lipid dysregulation.2026
15. CRISPR-Cas9-mediated upregulation of utrophin ameliorates Duchenne muscular dystrophyMolecular Therapy · 2026
16. Identification of alterations of immunometabolism associated with Pompe diseaseCellular and Molecular Life Sciences · 2026
17. Induced pluripotent stem cells and the rise of human-relevant in vitro models: promise, pitfalls, and the path forwardCell and Gene Therapy Insights · 2026
18. Ocular gene therapy mediated by AAV vectors: an overview of immunosuppressive treatments and immunomonitoring of patients involved in clinical trialsProgress in Retinal and Eye Research · 2026
19. Muscle meets Lysosomes: emerging strategies in muscular dystrophyAutophagy · 2026
20. A new dystrophin deficient rat model mirroring exon skipping in patients with DMD exon 45 deletionsDisease Models & Mechanisms · 2026