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弗里德里希共济失调 iPSC 来源感觉神经元在成年背根神经节移植中的体内存活和分化。

In vivo survival and differentiation of Friedreich ataxia iPSC-derived sensory neurons transplanted in the adult dorsal root ganglia.

机构信息

Department of Biomedical Engineering, The University of Melbourne, Parkville, Australia.

The Florey Institute of Neuroscience and Mental Health, Parkville, Australia.

出版信息

Stem Cells Transl Med. 2021 Aug;10(8):1157-1169. doi: 10.1002/sctm.20-0334. Epub 2021 Mar 18.

DOI:10.1002/sctm.20-0334
PMID:33734599
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC8284774/
Abstract

Friedreich ataxia (FRDA) is an autosomal recessive disease characterized by degeneration of dorsal root ganglia (DRG) sensory neurons, which is due to low levels of the mitochondrial protein Frataxin. To explore cell replacement therapies as a possible approach to treat FRDA, we examined transplantation of sensory neural progenitors derived from human embryonic stem cells (hESC) and FRDA induced pluripotent stem cells (iPSC) into adult rodent DRG regions. Our data showed survival and differentiation of hESC and FRDA iPSC-derived progenitors in the DRG 2 and 8 weeks post-transplantation, respectively. Donor cells expressed neuronal markers, including sensory and glial markers, demonstrating differentiation to these lineages. These results are novel and a highly significant first step in showing the possibility of using stem cells as a cell replacement therapy to treat DRG neurodegeneration in FRDA as well as other peripheral neuropathies.

摘要

弗里德赖希共济失调（FRDA）是一种常染色体隐性疾病，其特征是背根神经节（DRG）感觉神经元退化，这是由于线粒体蛋白 Frataxin 水平低所致。为了探索细胞替代疗法作为治疗 FRDA 的一种可能方法，我们研究了源自人胚胎干细胞（hESC）和 FRDA 诱导多能干细胞（iPSC）的感觉神经祖细胞移植到成年啮齿动物 DRG 区域。我们的数据显示，hESC 和 FRDA iPSC 衍生祖细胞分别在移植后 2 周和 8 周在 DRG 中存活和分化。供体细胞表达神经元标记物，包括感觉和神经胶质标记物，表明向这些谱系分化。这些结果是新颖的，并且是一个非常重要的第一步，表明使用干细胞作为细胞替代疗法治疗 FRDA 以及其他周围神经病中的 DRG 神经退行性变的可能性。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/e5e6/8284774/f2ebe4927fee/SCT3-10-1157-g005.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/e5e6/8284774/036d3af23ba2/SCT3-10-1157-g008.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/e5e6/8284774/244d06d79946/SCT3-10-1157-g003.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/e5e6/8284774/c700228556bb/SCT3-10-1157-g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/e5e6/8284774/e2f829784156/SCT3-10-1157-g007.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/e5e6/8284774/97a7878ad1b2/SCT3-10-1157-g006.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/e5e6/8284774/eaaed4a5ffcd/SCT3-10-1157-g004.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/e5e6/8284774/f2ebe4927fee/SCT3-10-1157-g005.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/e5e6/8284774/036d3af23ba2/SCT3-10-1157-g008.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/e5e6/8284774/244d06d79946/SCT3-10-1157-g003.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/e5e6/8284774/c700228556bb/SCT3-10-1157-g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/e5e6/8284774/e2f829784156/SCT3-10-1157-g007.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/e5e6/8284774/97a7878ad1b2/SCT3-10-1157-g006.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/e5e6/8284774/eaaed4a5ffcd/SCT3-10-1157-g004.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/e5e6/8284774/f2ebe4927fee/SCT3-10-1157-g005.jpg

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