Evaluation of Antisense Oligonucleotides Targeting ATXN3 in SCA3 Mouse Models

The most common dominantly inherited ataxia, spinocerebellar ataxia type 3 (SCA3), is an incurable neurodegenerative disorder caused by a CAG repeat expansion in the ATXN3 gene that encodes an abnormally long polyglutamine tract in the disease protein, ATXN3. Mice lacking ATXN3 are phenotypically no...

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Bibliografische gegevens
Gepubliceerd in:Mol Ther Nucleic Acids
Hoofdauteurs: Moore, Lauren R., Rajpal, Gautam, Dillingham, Ian T., Qutob, Maya, Blumenstein, Kate G., Gattis, Danielle, Hung, Gene, Kordasiewicz, Holly B., Paulson, Henry L., McLoughlin, Hayley S.
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: American Society of Gene & Cell Therapy 2017
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Original Article
Online toegang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5415970/
https://ncbi.nlm.nih.gov/pubmed/28624196
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtn.2017.04.005
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