Peptidic tools applied to redirect alternative splicing events.

摘要: Peptides are versatile and attractive biomolecules that can be applied to modulate genetic mechanisms like alternative splicing. In this process, a single transcript yields different mature RNAs leading the production of protein isoforms with diverse or even antagonistic functions. During splicing events, errors caused either by mutations present in genome defects imbalances regulatory factors. any case, have been related several diseases including muscular dystrophy, Alzheimer's disease cancer from almost every origin. One most effective approaches redirect events has attach cell-penetrating peptides oligonucleotides event restore correct gene expression. Here, we summarize how natural existing bioengineered over last few years regulate Under cellular backgrounds, shown function as potent vehicles for splice correction, their therapeutic benefits reached clinical trials patenting stages, emphasizing use an exciting tool treatment diseases.