Haplotype editing with CRISPR/Cas9 as a therapeutic approach for dominant-negative missense mutations in NEFL
PH Dua, BMJ Simon, CBE Marley, CM Feliciano… - bioRxiv, 2024 - biorxiv.org
Inactivation of disease alleles by allele-specific editing is a promising approach to treat
dominant-negative genetic disorders, provided the causative gene is haplo-sufficient. We …
dominant-negative genetic disorders, provided the causative gene is haplo-sufficient. We …