Early targeting of endoneurial macrophages alleviates the neuropathy and affects abnormal Schwann cell differentiation in a mouse model of Charcot‐Marie‐Tooth 1A
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DOI: 10.1002/glia.24158
Abstract: We have previously shown that targeting endoneurial macrophages with the orally applied CSF‐1 receptor specific kinase (c‐FMS) inhibitor PLX5622 from the age of 3 months onwards led to a substantial alleviation of the neuropathy in mouse… read more here.
Keywords: charcot marie; cmt1a; model; endoneurial macrophages ... See more keywords
Characterization of Charcot–Marie–Tooth optic neuropathy
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DOI: 10.1007/s00415-017-8645-2
Abstract: Varying degrees of optic neuropathy can be seen in patients with Charcot–Marie–Tooth (CMT) disease. To define and characterize the extent of optic neuropathy in patients with CMT2A and CMT1A, two patients from both sub-classifications were… read more here.
Keywords: cmt1a; patients cmt2a; charcot marie; optic neuropathy ... See more keywords
Targeting myelin lipid metabolism as a potential therapeutic strategy in a model of CMT1A neuropathy
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DOI: 10.1038/s41467-018-05420-0
Abstract: In patients with Charcot–Marie–Tooth disease 1A (CMT1A), peripheral nerves display aberrant myelination during postnatal development, followed by slowly progressive demyelination and axonal loss during adult life. Here, we show that myelinating Schwann cells in a… read more here.
Keywords: cmt1a; myelin lipid; model cmt1a; potential therapeutic ... See more keywords
Squalenoyl siRNA PMP22 nanoparticles are effective in treating mouse models of Charcot-Marie-Tooth disease type 1 A
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DOI: 10.1038/s42003-021-01839-2
Abstract: Charcot-Marie-Tooth disease type 1 A (CMT1A) lacks an effective treatment. We provide a therapy for CMT1A, based on siRNA conjugated to squalene nanoparticles (siRNA PMP22-SQ NPs). Their administration resulted in normalization of Pmp22 protein levels,… read more here.
Keywords: cmt1a; sirna pmp22; mouse models; disease ... See more keywords