Emerging Gene Correction Strategies for Muscular Dystrophies : Scientific Progress and Regulatory Impact

@inproceedings{Bachtarzi2018EmergingGC,
  title={Emerging Gene Correction Strategies for Muscular Dystrophies : Scientific Progress and Regulatory Impact},
  author={Houria Bachtarzi},
  year={2018}
}
Muscular dystrophies comprise a heterogeneous cluster of inherited muscle degenera‐ tive disorders with the common feature of progressive muscle weakness. These represent good candidates for treatment with gene-based therapies. Progress in gene transfer technologies has raised hopes for successful therapeutic restoration of mutated genes such as dystrophin… CONTINUE READING