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Pathogenic Mechanisms and Therapy Development for C9orf72 Amyotrophic Lateral Sclerosis/Frontotemporal Dementia
In 2011, a hexanucleotide repeat expansion in the first intron of the C9orf72 gene was identified as the most common genetic cause of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). The proposed disease mechanisms include loss of C9orf72 function and gain of toxicity from the...
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| Published in: | Neurotherapeutics |
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| Main Authors: | , |
| Format: | Artigo |
| Language: | Inglês |
| Published: |
Springer International Publishing
2019
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| Subjects: | |
| Online Access: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6985338/ https://ncbi.nlm.nih.gov/pubmed/31667754 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s13311-019-00797-2 |
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