Article ID | Journal | Published Year | Pages | File Type |
---|---|---|---|---|
8348111 | Peptides | 2015 | 11 Pages |
Abstract
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 to the production of protein isoforms with diverse or even antagonistic functions. During splicing events, errors can be caused either by mutations present in the genome or by defects or imbalances in regulatory protein factors. In any case, defects in alternative splicing have been related to several genetic diseases including muscular dystrophy, Alzheimer's disease and cancer from almost every origin. One of the most effective approaches to redirect alternative splicing events has been to attach cell-penetrating peptides to oligonucleotides that can modulate a single splicing event and restore correct gene expression. Here, we summarize how natural existing and bioengineered peptides have been applied over the last few years to regulate alternative splicing and genetic expression. Under different genetic and cellular backgrounds, peptides have been shown to function as potent vehicles for splice correction, and their therapeutic benefits have reached clinical trials and patenting stages, emphasizing the use of regulatory peptides as an exciting therapeutic tool for the treatment of different genetic diseases.
Keywords
SCOISSiAsBNPDMDNLSGFRESELMNsnRNAintronic splicing silencerOligonucleotidesPenetratinSplice-switching oligonucleotidessnRNPPPMOintronic splicing enhancersmall nuclear ribonucleic acidESSCPPmRNACBDANPPIDPMOPNAPTDSSOASOISEAntisense oligonucleotidesSmall interfering RNAsiRNATransportanNucleic acidsPeptide nucleic acidexonic splicing enhancerPrimary immunodeficiency diseasemutationsPENCalmodulin binding domainProtein transduction domainsDuchenne muscular dystrophynuclear localization signalLaminGlomerular filtration rateexonic splicing silenceratrial natriuretic peptideCell-penetrating peptideB-type natriuretic peptideCell penetrating peptidesSplicing
Related Topics
Life Sciences
Biochemistry, Genetics and Molecular Biology
Biochemistry
Authors
MartÃnez-Montiel Nancy, Rosas-Murrieta Nora, MartÃnez-Contreras Rebeca,