Innovation thérapeutique : de la physiopathologie à l’appliqué pour les pathologies neuromusculaires, la reproduction et le développement

Panels ERC déclarés au RNSR

Mots-clés des publications HAL repérées

Publications HAL récentes repérées

• Systemic distribution of tricyclo-DNA antisense oligonucleotide following intratracheal instillation in the mouse · 2026

  Résumé de la publication : This study evaluated intratracheal (IT) instillation as an alternative route for systemic delivery of therapeutic tricyclo-DNA antisense oligonucleotides (tcDNA-ASOs), using the Duchenne muscular dystrophy (DMD)-targeting compound SQY51 as a model. IT administration was compared with intravenous (IV) injection by assessing pharmacokinetics, biodistribution,…

• Efficient Downregulation of Flt-1 Mediated by Splice Switching ASO in Murine Endothelial Cells · 2026

  Résumé de la publication : Impaired angiogenesis is a common feature of several pathological conditions, including neuromuscular disorders. Such vascular defects not only contribute to disease progression but also may compromise the efficacy of systemically delivered therapies such as antisense oligonucleotides (ASOs) and adeno-associated virus vectors. Enhancing…

• Systemic distribution of tricyclo-DNA antisense oligonucleotide following intratracheal instillation in the mouse · 2026

  Résumé de la publication : Abstract This study evaluated intratracheal (IT) instillation as an alternative route for systemic delivery of therapeutic tricyclo-DNA antisense oligonucleotides (tcDNA-ASOs), using the Duchenne muscular dystrophy (DMD)-targeting compound SQY51 as a model. IT administration was compared with intravenous (IV) injection by assessing pharmacokinetics,…

• Residual Dp71 Expression Is Sufficient to Preserve Retinal Vascular Homeostasis in a Mouse Model of Duchenne Muscular Dystrophy · 2026

  Résumé de la publication : The dystrophin gene encodes multiple dystrophin isoforms with tissue-specific functions, including several shorter isoforms expressed in the central nervous system and retina. While Duchenne muscular dystrophy (DMD) has historically been characterized as a primary myopathy resulting from loss of the full-length dystrophin…

• First-in human assessment of the UroActive® electronic Artificial Urinary Sphincter for treating male stress urinary incontinence (SUI) · 2026

  Résumé de la publication : Purpose: To evaluate the feasibility, safety, and efficacy during the first year of use of the UroActive® device in males with stress urinary incontinence (SUI). Materials and Methods: This prospective, multicenter, single-arm, first-in-human study enrolled six male patients with bothersome SUI unresponsive…