Editorial: Genome and transcriptome editing to understand and treat neuromuscular diseases.

Frontiers in genome editing(2023)

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摘要
Neuromuscular diseases such as Duchenne muscular dystrophy and facioscapulohumeral muscular 14 dystrophy are debilitating conditions that affect millions of individuals worldwide. In recent years, 15 there has been a growing interest in the use of genome and transcriptome editing techniques to 16 understand and treat these diseases. This research topic brings together four articles that highlight the 17 latest advances in this field. 18 19The these diseases can be considered for the first time. They also highlight that the lack of effective 67 therapy for muscular dystrophies can be explained by the fact that more than 40 genes have been 68described to be involved in these diseases, resulting in a wide range of abnormalities and with the 69 large size of the mutated genes, it challenges classical gene replacement therapies. 70Overall, these articles demonstrate the potential of genome and transcriptome editing techniques to 71 improve the understanding and treatment of neuromuscular diseases. With the rapid pace of 72 technological advancements in this field, it is likely that we will see even more exciting 73 developments in the near future. 74
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关键词
5′UTR,Duchenne muscular dystrophy,GNE,SOD1,amyotrophic lateral sclerosis,antisense oligonucleotides,microRNA,shRNAs
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