CRISPR Gene Editing for Nucleotide Repeat Expansion Disorders: A Systematic Review of Preclinical and Clinical Evidence - PubMed
6 hours ago
- #NREDs
- #Gene Editing
- #CRISPR
- CRISPR-Cas gene editing shows promise for treating nucleotide repeat expansion disorders (NREDs) like Duchenne muscular dystrophy (DMD), Huntington's disease (HD), and myotonic dystrophy type 1 (DM1).
- A systematic review of 24 studies found CRISPR-mediated editing led to genomic correction, transcript rescue, and protein restoration in patient-derived cell models.
- Streptococcus pyogenes CRISPR-associated protein 9 (Cas9) was the most commonly used nuclease, with viral and nonviral delivery methods employed.
- Functional improvements included splicing correction and dystrophin restoration, but methodological flaws like lack of blinding and incomplete off-target reporting were noted.
- Further research is needed to address safety, methodological robustness, and in vivo validation before clinical translation.