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Molecular Biology

Molecular biology deals with nucleic acids and gene expression: reading, modifying and controlling genetic information to understand a biological function or correct an abnormality. Genome editing, RNA sequencing and gene therapy have made it one of the most dynamic engines of biomedical research.

It is the approach Inovarion draws on most. RNA sequencing (RNA-seq) is ubiquitous here, measuring gene expression at the scale of the transcriptome. CRISPR-Cas9 genome editing and CRISPRi interference, including in the form of genome-wide screens, make it possible to systematically validate the function of a target. AAV vectors serve both as expression tools and as gene-therapy vehicles. The generation of genetically modified models — knock-out, knock-in, transgenic, double knock-out — relies on cloning, mutagenesis, qPCR and RT-qPCR, and reporter genes such as secreted nanoluciferase. Finally, the study of gene regulation mobilises chromatin analysis: ChIP-seq, ATAC-seq, CUT&Tag and 4C-seq.

These tools underpin a large share of the corpus’s results. The laboratory took part in validating targets by CRISPR-Cas9 screening, such as the methyltransferase SETDB1 in uveal melanoma[2], and in dissecting the regulation of the DLK1/DIO3 locus by a β-catenin-dependent enhancer in liver tumorigenesis[4]. On the therapeutic side, AAV vectors have carried gene-therapy approaches in haemophilia, Duchenne dystrophy[1] and von Willebrand disease. Transcriptomics and genetically modified models also feed into almost every area, from oncology to metabolic diseases. This work has appeared in journals such as Molecular Therapy and Nucleic Acids Research.

Molecular biology is also the natural starting point of bioinformatic analysis: the sequencing data it generates are then decoded by Inovarion’s bioinformatics teams. This continuity, from bench to analysis, is one of the strengths of Inovarion’s model. Genome editing, sequencing and vectorology thus support partners’ projects, from target validation to therapeutic proof of concept.

See also: Single-cell RNA-seq ; “Bulk” RNA-seq & differential transcriptomics ; CRISPR & functional genomics ; ChIP-seq & epigenomics.

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Representative publications

  1. Forand et al. Long-Term Dystrophin Replacement Therapy in Duchenne Muscular Dystrophy Causes Cardiac Inflammation. JACC Basic Transl Sci, 2025. Record → · PubMed
  2. Krossa et al. SETDB1 is critically required for uveal melanoma growth and represents a promising therapeutic target. Cell Death & Disease, 2025. 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. Sanceau et al. DLK1/DIO3 locus upregulation by a β-catenin-dependent enhancer drives cell proliferation and liver tumorigenesis. Molecular Therapy, 2024. Record → · PubMed
  5. Traoré et al. GDF5 as a rejuvenating treatment for age-related neuromuscular failure. Brain, 2024. Record → · PubMed
  6. Hidaoui et al. Targeting heterochromatin eliminates chronic myelomonocytic leukemia malignant stem cells through reactivation of retroelements and immune pathways. Commun Biol, 2024. Record → · PubMed
  7. Deschamps et al. CXCL8 secreted by immature granulocytes inhibits WT hematopoiesis in chronic myelomonocytic leukemia. J Clin Invest, 2024. 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. Chappert et al. Human anti-smallpox long-lived memory B cells are defined by dynamic interactions in the splenic niche and long-lasting germinal center imprinting. Immunity, 2022. 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