Disease Mechanisms and Therapeutic Approaches in <i>C9orf72</i> ALS-FTD
A hexanucleotide repeat expansion mutation in the first intron of <i>C9orf72</i> is the most common known genetic cause of amyotrophic lateral sclerosis and frontotemporal dementia. Since the discovery in 2011, numerous pathogenic mechanisms, including both loss and gain of function, hav...
| Main Authors: | , , |
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| Format: | Article |
| Language: | English |
| Published: |
MDPI AG
2021-05-01
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| Series: | Biomedicines |
| Subjects: | |
| Online Access: | https://www.mdpi.com/2227-9059/9/6/601 |