Skip to content

In vivo & preclinical models

In vivo experimentation remains a decisive step in translational research: it is at the scale of the whole organism that the efficacy, safety and physiological relevance of a therapeutic hypothesis are verified. It is the organism-scale counterpart of the work carried out in cell biology.

Inovarion deploys a broad range of preclinical models. Genetically modified mouse models — conditional and tissue-specific knock-outs, knock-ins, transgenics, double knock-outs — form the core. The laboratory notably uses humanised models, carrying human proteins: it thus contributed to a fully humanised mouse model of von Willebrand disease, a valuable platform for evaluating new therapeutic options[3]. Added to these are dedicated disease models (reference mouse models — mdx and double knock-out — for Duchenne muscular dystrophy, haemophilic and bleeding models, high-fat-diet-induced obesity, lithium-pilocarpine epilepsy), patient-derived xenografts and orthotopic models in oncology, and zebrafish for in vivo imaging and optogenetics.

These models serve the in vivo validation of targets and therapies across every area: oncology (xenografts, orthotopic models, malignant transformation in zebrafish[7]), haematology (haemophilic models, humanised von Willebrand), rare diseases (Duchenne dystrophy), metabolism (adipocyte-specific knock-outs), cardiology (BMP9/BMP10 models[11]) and neurology (epileptogenesis[1]). Their value lies in their realism: humanised and tissue-specific conditional models, often coupled with iPSCs and transcriptomics to link the organism’s phenotype to molecular mechanisms.

Inovarion designs and operates these models in continuity with the in vitro and bioinformatic work carried out upstream — the generation of genetic models belongs to molecular biology, their readout to imaging and bioinformatics. This continuity, from the model to its readout, serves partners’ projects, from in vivo proof of concept to drug-candidate evaluation.

Contact an expert · See all publications

Representative publications

  1. Dufour et al. Spatiotemporal transcriptomic mapping reveals region-specific glial activation and astrocyte shifts in epileptogenesis beyond the hippocampus. Acta Neuropathol Commun, 2026. PubMed
  2. Kervella et al. Simtuzumab Attenuates Loxl2-Mediated Extracellular Matrix Remodeling and Preserves Cardiac Function in LMNA Mutation-Induced Dilated Cardiomyopathy. Circ Heart Fail, 2026. PubMed
  3. McCluskey et al. A fully humanized von Willebrand disease type 1 mouse model as unique platform to investigate novel therapeutic options. Haematologica, 2025. Record → · PubMed
  4. Sefiane et al. Consistent clinical factor VIII equivalency is unlikely for non-factor therapies in hemophilic mice. Haematologica, 2025. Record → · PubMed
  5. Plaisier et al. Prefrontal gamma oscillations and fear extinction learning require early postnatal interneuron-oligodendroglia communication. Nature Communications, 2025. PubMed
  6. Sayegh et al. Defective autophagy in CD4 T cells drives liver fibrosis via type 3 inflammation. Nature Communications, 2025. Record → · PubMed
  7. Scerbo et al. In vivo targeted and deterministic single-cell malignant transformation. eLife, 2025. Record → · PubMed
  8. Shi et al. FGFR3 Mutational Activation Can Induce Luminal-like Papillary Bladder Tumor Formation and Favors a Male Sex Bias. European Urology, 2023. Record → · PubMed
  9. Agopian et al. GlcNAc is a mast-cell chromatin-remodeling oncometabolite that promotes systemic mastocytosis aggressiveness. Blood, 2021. Record → · PubMed
  10. Huang et al. The corepressors GPS2 and SMRT control enhancer and silencer remodeling via eRNA transcription during inflammatory activation of macrophages. Molecular Cell, 2021. Record → · PubMed
  11. Bouvard et al. Different cardiovascular and pulmonary phenotypes for single- and double-knock-out mice deficient in BMP9 and BMP10. Cardiovasc Res, 2022. Record → · PubMed