首页>
外国专利>
RNA TARGETING OF MUTATIONS VIA SUPPESSOR tRNAs AND DEAMINASES
RNA TARGETING OF MUTATIONS VIA SUPPESSOR tRNAs AND DEAMINASES
展开▼
机译:通过抑制性tRNA和脱氨酶进行突变的RNA靶向
展开▼
页面导航
摘要
著录项
相似文献
摘要
Aspects of the disclosure relate to a gene therapy approach for diseases, disorders, or conditions caused by mutation in the stop codon utilizing modified tRNA. At least 10-15% of all genetic diseases, including muscular dystrophy (e.g. Duchene muscular dystrophy), some cancers, beta thalassemia, Hurler syndrome, and cystic fibrosis, fall into this category. Not to be bound by theory, it is believed that this approach is safer than CRISPR approaches due to minimal off-target effects and the lack of genome level changes.
展开▼