| Autor: |
Jack C. Reidling, Aroa Relaño-Ginés, Sandra M. Holley, Joseph Ochaba, Cindy Moore, Brian Fury, Alice Lau, Andrew H. Tran, Sylvia Yeung, Delaram Salamati, Chunni Zhu, Asa Hatami, Carlos Cepeda, Joshua A. Barry, Talia Kamdjou, Alvin King, Dane Coleal-Bergum, Nicholas R. Franich, Frank M. LaFerla, Joan S. Steffan, Mathew Blurton-Jones, Charles K. Meshul, Gerhard Bauer, Michael S. Levine, Marie-Francoise Chesselet, Leslie M. Thompson |
| Jazyk: |
angličtina |
| Rok vydání: |
2018 |
| Předmět: |
|
| Zdroj: |
Stem Cell Reports, Vol 10, Iss 1, Pp 58-72 (2018) |
| Druh dokumentu: |
article |
| ISSN: |
2213-6711 |
| DOI: |
10.1016/j.stemcr.2017.11.005 |
| Popis: |
Summary: Huntington's disease (HD) is an inherited neurodegenerative disorder with no disease-modifying treatment. Expansion of the glutamine-encoding repeat in the Huntingtin (HTT) gene causes broad effects that are a challenge for single treatment strategies. Strategies based on human stem cells offer a promising option. We evaluated efficacy of transplanting a good manufacturing practice (GMP)-grade human embryonic stem cell-derived neural stem cell (hNSC) line into striatum of HD modeled mice. In HD fragment model R6/2 mice, transplants improve motor deficits, rescue synaptic alterations, and are contacted by nerve terminals from mouse cells. Furthermore, implanted hNSCs are electrophysiologically active. hNSCs also improved motor and late-stage cognitive impairment in a second HD model, Q140 knockin mice. Disease-modifying activity is suggested by the reduction of aberrant accumulation of mutant HTT protein and expression of brain-derived neurotrophic factor (BDNF) in both models. These findings hold promise for future development of stem cell-based therapies. |
| Databáze: |
Directory of Open Access Journals |
| Externí odkaz: |
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