Haplotype editing with CRISPR-Cas9 as a therapeutic approach for dominant-negative missense mutations in NEFL.

Dua PH, Simon BMJ, Marley CBE, Feliciano CM, Watry HL, Cowan QT, Steury D, Abraham A, Gilbertson EN, Ramey GD, Capra JA, Conklin BR, Judge LM