Title |
Modulation of aberrant splicing in human RNA diseases by chemical compounds
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Published in |
Human Genetics, March 2017
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DOI | 10.1007/s00439-017-1789-4 |
Pubmed ID | |
Authors |
Naoyuki Kataoka |
Abstract |
Pre-mRNA splicing is an essential step for gene expression in higher eukaryotes. Alternative splicing contributes to diversity of the expressed proteins from the limited number of genes. Disruption of splicing regulation often results in hereditary and sporadic diseases called as 'RNA diseases'. Modulation of splicing by small chemical compounds and nucleic acids has been tried to target aberrant splicing in those diseases. Several RNA diseases and splicing-target therapeutic approaches will be briefly introduced in this review. Accumulating knowledge about molecular mechanism of aberrant splicing and their correction by chemical compounds is important not only for RNA biologists, but also for clinicians who desire therapies for those diseases. |
Mendeley readers
Geographical breakdown
Country | Count | As % |
---|---|---|
Unknown | 40 | 100% |
Demographic breakdown
Readers by professional status | Count | As % |
---|---|---|
Student > Ph. D. Student | 9 | 23% |
Researcher | 9 | 23% |
Student > Master | 8 | 20% |
Student > Bachelor | 2 | 5% |
Professor | 2 | 5% |
Other | 2 | 5% |
Unknown | 8 | 20% |
Readers by discipline | Count | As % |
---|---|---|
Biochemistry, Genetics and Molecular Biology | 10 | 25% |
Agricultural and Biological Sciences | 10 | 25% |
Medicine and Dentistry | 3 | 8% |
Chemistry | 2 | 5% |
Nursing and Health Professions | 1 | 3% |
Other | 3 | 8% |
Unknown | 11 | 28% |