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DM1 Phenotype Variability and Triplet Repeat Instability: Challenges in the Development of New Therapies
Myotonic dystrophy type 1 (DM1) is a complex neuromuscular disease caused by an unstable cytosine thymine guanine (CTG) repeat expansion in the DMPK gene. This disease is characterized by high clinical and genetic variability, leading to some difficulties in the diagnosis and prognosis of DM1. Bette...
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| Yayımlandı: | Int J Mol Sci |
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| Asıl Yazarlar: | , |
| Materyal Türü: | Artigo |
| Dil: | Inglês |
| Baskı/Yayın Bilgisi: |
MDPI
2020
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| Konular: | |
| Online Erişim: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7014087/ https://ncbi.nlm.nih.gov/pubmed/31936870 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/ijms21020457 |
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