Publications by authors named "Romains Joubert"
Article Synopsis
- Facioscapulohumeral dystrophy (FSHD) is the most prevalent adult muscle disorder, characterized by the harmful expression of the DUX4 protein, which causes muscle cell death.
- Researchers aimed to permanently disable DUX4 gene expression using gene editing tools like TALEN and CRISPR-Cas9 on affected muscle cells, but only a small percentage of the edits were successful.
- Findings revealed that even when the gene's poly(A) signal was removed, DUX4 mRNA continued to be produced due to alternative signals, indicating that this gene editing approach may not be an effective treatment for FSHD.
