Alternative splicing aberrations can cause disease or promote its progression, including the development of cancer. Reversal of pathologic splicing events is a potential therapy for those cases. Efficient methods for identification and characterization of small molecule modulators of specific splicing events ...
The small molecule acts as a specific splicing enhancer cooperatively with the splicing regulatory network. Our investigations uncovered a novel concept for gene-specific alternative splicing correction that we coined 5 splice site bulge repair.
Conclusions This is the first demonstration that moderation of splicing factor levels is associated with reversal of cellular senescence in human primary fibroblasts. Small molecule modulators of such targets may therefore represent promising novel anti-degenerative therapies.Back...
This is the first demonstration that moderation of splicing factor levels is associated with reversal of cellular senescence in human primary fibroblasts. Small molecule modulators of such targets may therefore represent promising novel anti-degenerative therapies. The online version of this article (...
Identification of Small Molecule Inhibitors of Pre-mRNA Splicing Background: There is a need for new small molecule Pawellek,Andrea,McElroy,... - 《Journal of Biological Chemistry》 被引量: 22发表: 2014年 A High-Throughput Assay to Identify Small-Molecule Modulators of Alternative Pre-mRNA Spli...
In recent years, several high-throughput screening studies have been carried out to identify small molecule modulators of general or alternative splicing in proliferating cells30,31. For example, one approach has shown that indole derivatives are potent splicing inhibitors with a selective action on sp...
for identifying small molecule compounds targeting m6A regulators. For example, based on the substrate analogues adenosine of the m6A regulator, the corresponding competitive inhibitor, such as METTL3 inhibitor UZH1a [12], was designed by virtual screening. Based on the m6A competition assay for the...
Furthermore, we examine potential small molecule drug and gene therapies for the treatment of AS and associated challenges faced by the therapeutic design. Finally, gene therapy offers the opportunity for precision medicine in AS and advancements in the treatment of this disorder can serve as a ...
small molecule-based therapeutics may have the advantage over others to be able to overcome the hurdles of the blood–brain barrier, poor absorption, and allergic reactions. In this review, we have summarized various TREDs and envisage the potential of small molecule-based therapeutics for targeting...
9e), which indicates that LINP1 is a component of the abnormal DNA-PK complex formed in the absence of scaRNA2. In contrast, depletion of LINP1 elevated the binding of scaRNA2 to pDNA-PKcs (Fig. 6g) and lessened the association between DNA-PKcs and Ku70/80 (Fig. 6h). This is ...