A5012359607- Genetic Neurodegenerative Diseases
- Mitochondrial Function and Pathology
- T-cell and Retrovirus Studies
- Vector-Borne Animal Diseases
- Animal Disease Management and Epidemiology
- CRISPR and Genetic Engineering
- Muscle Physiology and Disorders
Centre de Recherche en Myologie
2019-2023
University of Liège
2019-2023
Sorbonne Université
2019-2023
Inserm
2019-2023
Institut de Myologie
2023
Gembloux Agro-Bio Tech
2020
Myotonic dystrophy type 1 (DM1) is a neuromuscular disease that originates from an expansion of CTG microsatellites in the 3' untranslated region DMPK gene, thus leading to expression transcripts containing expanded CUG repeats (CUGexp). The pathophysiology explained by toxic RNA gain function where CUGexp RNAs form nuclear aggregates sequester and alter MBNL splicing factors, triggering misregulation linked DM1 symptoms. There currently no cure for DM1, most therapeutic strategies aim at...
Key Points By disrupting basal transcription machinery, HBZ RNA inhibits sense of human T-cell leukemia virus type 1. Repression genomic expression may allow entry into proviral latency and escape from immune response.