GeneticsMolecular TherapyPeer-reviewed
Haplotype editing with CRISPR/Cas9 as a therapeutic approach for dominant-negative missense mutations in NEFL
Poorvi H. Dua, Bazilco M.J. Simon, Chiara B.E. Marley…·Luke M. Judge
This paper demonstrates haplotype editing with CRISPR/Cas9 to target NEFL missense mutations in iPSC-derived motor neurons, showing therapeutic potential and challenges in disrupting mutant allele expression.
1 citation