Cellular reprogramming as a new treatment for Charcot–Marie–Tooth disease

Publish Year: 1398
نوع سند: مقاله کنفرانسی
زبان: English
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BSIPD01_013

تاریخ نمایه سازی: 5 بهمن 1398

Abstract:

The genetic neuropathies are a heterogeneous group of disorders and can mostly be categorized into two groups: those in which the neuropathy is the sole or primary portion of the disease (Charcot–Marie–Tooth disease, CMT) and those in which the neuropathy is part of a more widespread neurologic or multisystem disease (e.g., familial amyloid polyneuropathy, neuropathies associated with mitochondrial diseases, with hereditary ataxias, porphyrias).Materials & Methods: This literature review is based on English-language articles sourced from PubMed without date limitation for using the following terms. Cellular reprogramming technique, Charcot Marie Tooth disease, Inherited peripheral disorder, Inherited peripheral neuropathies.Results: Cellular reprogramming and high throughput drug screening are two new treatment strategies for CMT. A cellular reprogramming technique is by generation of human neural crest progenitors derived from a patient with HSAN type III, the precursors of sensory and autonomic neurons, the cell types most affected by this condition.Conclusion & discussion: Finding compounds that at least partially rescued this phenotype, validating this platform for drug discovery in inherited neuropathies.

Authors

Mehrnaz Moattari

Department of Animal Biology, Faculty of Biological Science, Kharazmi University, Tehran, Iran

Farahnaz Moattari

Faculty of Agriculture and Natural Resources, Persian Gulf University, Bushehr, Iran

Gholamreza Kaka

Neuroscience Research Center, Baqiyatallah University of Medical Sciences, Tehran, Iran