Innovative Therapeutic and Delivery Approaches Using Nanotechnology to Correct Splicing Defects Underlying Disease
| Autor: | María Luján Cuestas, Cristina Hernández-Munain, Cristina Moreno-Castro, Marc Suñé-Pou, Josep M. Suñé-Negre, Carlos Suñé, María J. Limeres |
|---|---|
| Přispěvatelé: | Ministerio de Economía y Competitividad (España), European Commission |
| Jazyk: | angličtina |
| Rok vydání: | 2020 |
| Předmět: |
0301 basic medicine
GENE EDITING lcsh:QH426-470 SMaRT NANOPARTICLE Nanotechnology Review Disease Gene delivery Biology ASOs purl.org/becyt/ford/1 [https] Transcriptome 03 medical and health sciences splicing 0302 clinical medicine Gene therapy Genome editing Genetics purl.org/becyt/ford/1.6 [https] Gene Genetics (clinical) SMART Nanopartícules gene editing Nanotecnologia nanoparticle Alternative splicing gene therapy and therapeutic delivery SIRNAS ASOS siRNAs lcsh:Genetics 030104 developmental biology Underlying disease GENE THERAPY AND THERAPEUTIC DELIVERY 030220 oncology & carcinogenesis RNA splicing Molecular Medicine Teràpia genètica Nanoparticles RNA SPLICING |
| Zdroj: | Frontiers in Genetics, Vol 11 (2020) Dipòsit Digital de la UB Universidad de Barcelona Frontiers in Genetics CONICET Digital (CONICET) Consejo Nacional de Investigaciones Científicas y Técnicas instacron:CONICET |
| ISSN: | 1664-8021 |
| Popis: | Alternative splicing of pre-mRNA contributes strongly to the diversity of cell- and tissue-specific protein expression patterns. Global transcriptome analyses have suggested that >90% of human multiexon genes are alternatively spliced. Alterations in the splicing process cause missplicing events that lead to genetic diseases and pathologies, including various neurological disorders, cancers, and muscular dystrophies. In recent decades, research has helped to elucidate the mechanisms regulating alternative splicing and, in some cases, to reveal how dysregulation of these mechanisms leads to disease. The resulting knowledge has enabled the design of novel therapeutic strategies for correction of splicing-derived pathologies. In this review, we focus primarily on therapeutic approaches targeting splicing, and we highlight nanotechnology-based gene delivery applications that address the challenges and barriers facing nucleic acid-based therapeutics. This work was supported by grants from the Spanish Ministry of Economy and Competitiveness (grant number BFU2017-89179-R to CS and grant number BFU2016-79699-P to CH-M). Support from the European Region Development Fund (ERDF [FEDER]) is also acknowledged. |
| Databáze: | OpenAIRE |
| Externí odkaz: |
|