Use of human stem cells in Huntington disease modeling and translational research
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- 作者：Monika M. Golasa ; mgolas@biomed.au.dk" class="auth_mail" title="E-mail the corresponding author ; Bjoern Sanderb
- 关键词：AAV4 ; adeno-associated virus serotype 4 ; BDNF ; brain-derived neurotrophic factor ; bFGF ; fibroblast growth factor 2 ; BME ; β-mercaptoethanol ; BMP ; bone morphogenetic protein ; Cas9 ; CRISPR associated protein 9 ; Cla ; claustrum ; CN ; caudate nucleus ; CRISPR ; clustered regulatory interspaced short palindromic repeat ; D ; day ; DARPP32 ; dopamine- and cAMP-regulated phosphoprotein of 32 ; kDa ; db-cAMP ; dibutyryl-cAMP ; DKK-1 ; dickkopf-1 ; EGF ; epidermal growth factor ; ESC ; embryonic stem cell ; GABA ; γ-ami
- 刊名：Experimental Neurology
- 出版年：2016
- 出版时间：April 2016
- 年：2016
- 卷：278
- 期：Complete
- 页码：76-90
- 全文大小：3002 K
文摘
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Approaches to differentiate medium spiny neurons (MSN) from stem cells
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Neurons differentiated from stem cells recapitulate Huntington disease (HD) in vitro.
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Transplantation of fetal neural stem cells improves HD in a subset of patients.
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Induced pluripotent stem cells enable the use of patient-specific MSN progenitor cells.
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Genome editing to correct the HTT mutation in patient-specific stem cells