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

bioRxiv : the preprint server for biology

Dua PH, Simon BMJ, Marley CBE, Feliciano CM, Watry HL, Steury D, Abraham A, Gilbertson EN, Ramey GD, Capra JA, Conklin BR, Judge LM. Haplotype editing with CRISPR/Cas9 as a therapeutic approach for dominant-negative missense mutations in NEFL. bioRxiv. 2024 Dec 22.

2024
https://researcherprofiles.org/profile/579101760
39763989

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