Table 1.

The next good CMT targets for gene therapy development.

Gene/disease	Mechanism/approach	Patients/natural history	Animal model
MFN2/CMT2A	LoF/dominant negative, increased MFN2 or MFN1 expression	Most common axonal neuropathy, natural history available	Transgenic mouse models, rat knockin?
GDAP1/CMT4A, CMT2K	LoF, gene replacement	Natural history available	Knockout mice
FIG4/CMT4J	Recessive LoF, gene replacement	Rare, natural history limited	Pale Tremor Mouse
IGHMBP2/CMT2S, SMARD1	Recessive LoF, gene replacement	Rare, natural history limited, better for SMARD1	Nmd mouse
CNTNAP1/CHN3, LCCS7	Recessive LoF, gene replacement	Very rare, very limited natural history	Knockout and shambling mouse models