UM
Neural stem cells for disease modeling and evaluation of therapeutics for Tay-Sachs disease
Vu, Mylinh; Li, Rong; Baskfield, Amanda; Lu, Billy; Farkhondeh, Atena; Gorshkov, Kirill; Motabar, Omid; Beers, Jeanette; Chen, Guokai; Zou, Jizhong; Espejo-Mojica, Angela J.; Rodriguez-Lopez, Alexander; Almeciga-Diaz, Carlos J.; Barrera, Luis A.; Jiang, Xuntian; Ory, Daniel S.; Marugan, Juan J.; Zheng, Wei
2018-09-17
Source PublicationORPHANET JOURNAL OF RARE DISEASES
ISSN1750-1172
Volume13
AbstractBackground: Tay-Sachs disease (TSD) is a rare neurodegenerative disorder caused by autosomal recessive mutations in the HEXA gene on chromosome 15 that encodes beta-hexosaminidase. Deficiency in HEXA results in accumulation of GM2 ganglioside, a glycosphingolipid, in lysosomes. Currently, there is no effective treatment for TSD.
KeywordTay-Sachs disease Induced pluripotent stem cells Neural stem cells Cyclodextrin HP beta CD delta-tocopherol Enzyme replacement therapy Hexosaminidase A GM2 gangliosidosis High throughput screening Drug discovery
DOI10.1186/s13023-018-0886-3
URLView the original
Indexed BySCI
Language英语
WOS Research AreaGenetics & Heredity ; Research & Experimental Medicine
WOS SubjectGenetics & Heredity ; Medicine, Research & Experimental
WOS IDWOS:000444968000001
PublisherBMC
The Source to ArticleWOS
Fulltext Access
Citation statistics
Cited Times [WOS]:1   [WOS Record]     [Related Records in WOS]
Document TypeJournal article
CollectionUniversity of Macau
Recommended Citation
GB/T 7714
Vu, Mylinh,Li, Rong,Baskfield, Amanda,et al. Neural stem cells for disease modeling and evaluation of therapeutics for Tay-Sachs disease[J]. ORPHANET JOURNAL OF RARE DISEASES,2018,13.
APA Vu, Mylinh.,Li, Rong.,Baskfield, Amanda.,Lu, Billy.,Farkhondeh, Atena.,...&Zheng, Wei.(2018).Neural stem cells for disease modeling and evaluation of therapeutics for Tay-Sachs disease.ORPHANET JOURNAL OF RARE DISEASES,13.
MLA Vu, Mylinh,et al."Neural stem cells for disease modeling and evaluation of therapeutics for Tay-Sachs disease".ORPHANET JOURNAL OF RARE DISEASES 13(2018).
Related Services
Recommend this item
Bookmark
Usage statistics
Export to Endnote
Google Scholar
Similar articles in Google Scholar
[Vu, Mylinh]'s Articles
[Li, Rong]'s Articles
[Baskfield, Amanda]'s Articles
Baidu academic
Similar articles in Baidu academic
[Vu, Mylinh]'s Articles
[Li, Rong]'s Articles
[Baskfield, Amanda]'s Articles
Bing Scholar
Similar articles in Bing Scholar
[Vu, Mylinh]'s Articles
[Li, Rong]'s Articles
[Baskfield, Amanda]'s Articles
Terms of Use
No data!
Social Bookmark/Share
All comments (0)
No comment.
 

Items in the repository are protected by copyright, with all rights reserved, unless otherwise indicated.