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Allele-Specific Silencing of Mutant Huntingtin in Rodent Brain and Human Stem Cells
Huntington's disease (HD) is an autosomal dominant neurodegenerative disorder resulting from polyglutamine expansion in the huntingtin (HTT) protein and for which there is no cure. Although suppression of both wild type and mutant HTT expression by RNA interference is a promising therapeutic st...
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Main Authors: | , , , , , , , , , , , , , , , , , |
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Formato: | Artigo |
Idioma: | Inglês |
Publicado em: |
Public Library of Science
2014
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Assuntos: | |
Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4057216/ https://ncbi.nlm.nih.gov/pubmed/24926995 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0099341 |
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