| 摘要 |
This invention relates, e.g., to a method for enhancing exon skipping in a pre-mRNA of interest, comprising contacting the pre-mRNA with an effective amount of a small molecule selected from the compounds shown in Table 1, or a pharmaceutically acceptable salt, hydrate, solvate, or isomer thereof, and, optionally, with an antisense oligonucleotide that is specific for a splicing sequence in the pre-mRNA Methods for treating Duchenne muscular dystrophy (DMD) are disclosed. |
| 主权项 |
1. A method for enhancing exon skipping in an mRNA of interest, comprising contacting the mRNA with an effective amount of a small molecule compound selected from one or more of furaltadone hydrochloride, 5-iodotubericidin, bendroflumethiazide, cyclopiazonic acid, GW 5074, indirubin, rescinnamin, U-0126, acetopromazine maleate salt, Ro 31-8220, dantrolene, dichlorobenzamil, ellipticine, fenbendazole, GF 109203X, halofantrine, niclosamide, pimozide, reserpine, syringospine, Ryanodine, RyCal S107, piperacetazine, fluphenazine dihydrochloride, trifluorperazine dihydrochloride, yohimbinic acid, or menadione,
or a pharmaceutically acceptable salt, hydrate, solvate, or isomer thereof. |
