Table 5.
Treatment strategies in AR demyelinating neuropathies
| Gene | Treatment | Mechanism | Model | Reference |
|---|---|---|---|---|
| FIG4 | Ca2+ (Calcium) chelation | Reduction of abnormally elevated intracellular Ca2+ | Plt (Fig4-/-) mice | Hu (2018) |
| TRPML1 synthetic ligand | Reduction of abnormally elevated intra-lysosomal Ca2+ | Plt (Fig4-/-) mice culture cells | Zou (2015) | |
| AAV9-mediated FIG4 gene delivery | Restore FIG4 expression | Plt ( ig4-/-) mice | Presa (2021) | |
| MTMR | Niaspan | NRG1 type III inhibition (by activating TACE secretase) | Mtmr2-null mice | Bolino (2016) |
| PIKfyve kinase inhibitor (YM201636) | Reduction of PtdIns(3,5)P2 phospholipid levels |
Mtmr2-null Schwann cell/DRG neuron co-cultures |
Vaccari (2011) | |
| SH3TC2 | lentiviral vector LV-Mpz.SH3TC2.myc | Increase expression of the human SH3TC2 cDNA | Sh3tc2-/- mice | Schiza (2019) |
FIG4 FIG4 phosphoinositide 5-phosphatase, TRMPL1 transient receptor potential cation channel, mucolipin subfamily, AAV9 adeno-associated virus 9, MTMR myotubularin-related protein, NRG1 neuroregulin1, TACE tumor necrosis factor-α converting enzyme