stem cells

Pharmaceutical composition for preventing or treating Charcot-Marie-Tooth disease comprising insulin secreted from mesenchymal stem cells or mesenchymal stem cells

September 29, 2021

https://patents.google.com/patent/KR20210117771A/en

Pharmaceutical composition for preventing or treating Charcot-Marie-Tooth disease comprising insulin secreted from mesenchymal stem cells or mesenchymal stem cells
Abstract
The present invention relates to a pharmaceutical composition for preventing or treating Charcot-Marie-Tooth disease, comprising mesenchymal stem cells or insulin secreted from mesenchymal stem cells.

Optimized Protocol to Generate Spinal Motor Neuron Cells from Induced Pluripotent Stem Cells from Charcot Marie Tooth Patients

June 27, 2020

https://www.mdpi.com/2076-3425/10/7/407

https://www.ncbi.nlm.nih.gov/pubmed/32605002?dopt=Abstract

Optimized Protocol to Generate Spinal Motor Neuron Cells from Induced Pluripotent Stem Cells from Charcot Marie Tooth Patients.

Brain Sci. 2020 Jun 27;10(7):

Authors: Faye PA, Vedrenne N, Miressi F, Rassat M, Romanenko S, Richard L, Bourthoumieu S, Funalot B, Sturtz F, Favreau F, Lia AS

Abstract

Modelling rare neurogenetic diseases to develop new therapeutic strategies is highly challenging. The use of human-induced pluripotent stem cells (hiPSCs) is a powerful approach to obtain specialized cells from patients. For hereditary peripheral neuropathies, such as Charcot-Marie-Tooth disease (CMT) Type II, spinal motor neurons (MNs) are impaired but are very difficult to study. Although several protocols are available to differentiate hiPSCs into neurons, their efficiency is still poor for CMT patients. Thus, our goal was to develop a robust, easy, and reproducible protocol to obtain MNs from CMT patient hiPSCs. The presented protocol generates MNs within 20 days, with a success rate of 80%, using specifically chosen molecules, such as Sonic Hedgehog or retinoic acid. The timing and concentrations of the factors used to induce differentiation are crucial and are given hereby. We then assessed the MNs by optic microscopy, immunocytochemistry (Islet1/2, HB9, Tuj1, and PGP9.5), and electrophysiological recordings. This method of generating MNs from CMT patients in vitro shows promise for the further development of assays to understand the pathological mechanisms of CMT and for drug screening.

PMID: 32605002 [PubMed]

PubMed:32605002

Faye PA, Vedrenne N, Miressi F, Rassat M, Romanenko S, Richard L, Bourthoumieu S, Funalot B, Sturtz F, Favreau F, Lia AS

Differentiation of Human Tonsil-Derived Mesenchymal Stem Cells into Schwann-Like Cells Improves Neuromuscular Function in a Mouse Model of Charcot-Marie-Tooth Disease Type 1A

August 14, 2018

https://www.mdpi.com/1422-0067/19/8/2393

https://www.ncbi.nlm.nih.gov/pubmed/30110925?dopt=Abstract

Differentiation of Human Tonsil-Derived Mesenchymal Stem Cells into Schwann-Like Cells Improves Neuromuscular Function in a Mouse Model of Charcot-Marie-Tooth Disease Type 1A.

Int J Mol Sci. 2018 Aug 14;19(8):

Authors: Park S, Jung N, Myung S, Choi Y, Chung KW, Choi BO, Jung SC

Abstract

Charcot-Marie-Tooth disease type 1A (CMT1A) is the most common inherited motor and sensory neuropathy, and is caused by duplication of PMP22, alterations of which are a characteristic feature of demyelination. The clinical phenotype of CMT1A is determined by the degree of axonal loss, and patients suffer from progressive muscle weakness and impaired sensation. Therefore, we investigated the potential of Schwann-like cells differentiated from human tonsil-derived stem cells (T-MSCs) for use in neuromuscular regeneration in trembler-J (Tr-J) mice, a model of CMT1A. After differentiation, we confirmed the increased expression of Schwann cell (SC) markers, including glial fibrillary acidic protein (GFAP), nerve growth factor receptor (NGFR), S100 calcium-binding protein B (S100B), glial cell-derived neurotrophic factor (GDNF), and brain-derived neurotrophic factor (BDNF), which suggests the differentiation of T-MSCs into SCs (T-MSC-SCs). To test their functional efficiency, the T-MSC-SCs were transplanted into the caudal thigh muscle of Tr-J mice. Recipients’ improved locomotive activity on a rotarod test, and their sciatic function index, which suggests that transplanted T-MSC-SCs ameliorated demyelination and atrophy of nerve and muscle in Tr-J mice. Histological and molecular analyses showed the possibility of in situ remyelination by T-MSC-SCs transplantation. These findings demonstrate that the transplantation of heterologous T-MSC-SCs induced neuromuscular regeneration in mice and suggest they could be useful for the therapeutic treatment of patients with CMT1A disease.

PMID: 30110925 [PubMed – indexed for MEDLINE]

PubMed:30110925

Park S, Jung N, Myung S, Choi Y, Chung KW, Choi BO, Jung SC