| 摘要 |
The present invention provides a method of preventing a splicing event in a pre-mRNA molecule, comprising contacting the pre-mRNA and/or elements of the splicing machinery with a small molecule compound identified according to th e methods described herein to prevent the splicing event in the pre-mRNA molecule. Further provided is a method of inducing a splicing event in a pre - mRNA molecule, comprising contacting the pre-mRNA and/or elements of the splicing machinery with a small molecule compound identified according to th e methods described herein to induce the splicing event in the pre-mRNA molecule. Furthermore, a method is provided herein of treating a patient having a disorder associated with an alternative or aberrant splicing event in a pre-mRNA molecule, comprising administering to the patient a therapeutical ly effective amount of a compound identified according to the methods described herein to prevent an alternative or aberrant splicing event in a pre-mRNA molecule, thereby treating the patient.
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