Inducible expression of human C9ORF72 36x G4C2 hexanucleotide repeats is sufficient to cause RAN translation and rapid muscular atrophy in mice [RESEARCH ARTICLE]
In conclusion, expression of 36x pure G4C2 repeats including 100-bp human flanking regions is sufficient for RAN translation of sense DPRs, and evokes a functional locomotor phenotype. Our inducible mouse model suggests that early diagnosis and treatment are important for C9FTD/ALS patients.
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Source: DMM Disease Models and Mechanisms - Category: Biomedical Science Authors: Riemslagh, F. W., van der Toorn, E. C., Verhagen, R. F. M., Maas, A., Bosman, L. W. J., Hukema, R. K., Willemsen, R. Tags: Neurodegenerative disorders, Neuromuscular RESEARCH ARTICLE Source Type: research
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