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- W2318486985 endingPage "924" @default.
- W2318486985 startingPage "914" @default.
- W2318486985 abstract "Alternative splicing is a critical step where a limited number of human genes generate a complex and diverse proteome. Various diseases, including inherited diseases with abnormalities in the “genome code,” have been found to result in an aberrant mis-spliced “transcript code” with correlation to the resulting phenotype. Chemical compound-based and nucleic acid-based strategies are trying to target this mis-spliced “transcript code”. We will briefly mention about how to obtain splicing-modifying-compounds by high-throughput screening and overview of what is known about compounds that modify splicing pathways. The main focus will be on RNA-binding protein kinase inhibitors. In the main text, we will refer to diseases where splicing-modifying-compounds have been intensively investigated, with comparison to nucleic acid-based strategies. The information on their involvement in mis-splicing as well as nonsplicing events will be helpful in finding better compounds with less off-target effects for future implications in mis-splicing therapy." @default.
- W2318486985 created "2016-06-24" @default.
- W2318486985 creator A5022179217 @default.
- W2318486985 creator A5090303297 @default.
- W2318486985 date "2015-01-20" @default.
- W2318486985 modified "2023-10-16" @default.
- W2318486985 title "Modulation of Alternative Splicing with Chemical Compounds in New Therapeutics for Human Diseases" @default.
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