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Investigational Assay for Haplotype Phasing of the Huntingtin Gene
Novel treatments for Huntington’s disease (HD), a progressive neurodegenerative disorder, include selective targeting of the mutant allele of the huntingtin gene (mHTT) carrying the abnormally expanded disease-causing cytosine-adenine-guanine (CAG) repeat. WVE-120101 and WVE-120102 are investigation...
Αποθηκεύτηκε σε:
| Τόπος έκδοσης: | Mol Ther Methods Clin Dev |
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| Κύριοι συγγραφείς: | , , , , , , , , , , , |
| Μορφή: | Artigo |
| Γλώσσα: | Inglês |
| Έκδοση: |
American Society of Gene & Cell Therapy
2020
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| Θέματα: | |
| Διαθέσιμο Online: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7648085/ https://ncbi.nlm.nih.gov/pubmed/33209959 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2020.09.003 |
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