Associated Papers
Haplotype editing with CRISPR-Cas9 as a therapeutic approach for dominant-negative missense mutations in NEFL.
Dua PH et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy, 2025
A novel recessive Nefl mutation causes a severe, early-onset axonal neuropathy.
Yum SW et al.
Annals of Neurology, 2009
Charcot-Marie-Tooth disease type 2E, a disorder of the cytoskeleton.
Fabrizi GM et al.
Brain : a Journal of Neurology, 2007
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