RNA Trans-Splicing Modulation via Antisense Molecule Interference.
Int J Mol Sci
; 19(3)2018 Mar 07.
Article
em En
| MEDLINE
| ID: mdl-29518954
ABSTRACT
In recent years, RNA trans-splicing has emerged as a suitable RNA editing tool for the specific replacement of mutated gene regions at the pre-mRNA level. Although the technology has been successfully applied for the restoration of protein function in various genetic diseases, a higher trans-splicing efficiency is still desired to facilitate its clinical application. Here, we describe a modified, easily applicable, fluorescence-based screening system for the generation and analysis of antisense molecules specifically capable of improving the RNA reprogramming efficiency of a selected KRT14-specific RNA trans-splicing molecule. Using this screening procedure, we identified several antisense RNAs and short rationally designed oligonucleotides, which are able to increase the trans-splicing efficiency. Thus, we assume that besides the RNA trans-splicing molecule, short antisense molecules can act as splicing modulators, thereby increasing the trans-splicing efficiency to a level that may be sufficient to overcome the effects of certain genetic predispositions, particularly those associated with dominantly inherited diseases.
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Texto completo:
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Base de dados:
MEDLINE
Assunto principal:
Splicing de RNA
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Regulação da Expressão Gênica
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Oligonucleotídeos Antissenso
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Trans-Splicing
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Interferência de RNA
Limite:
Humans
Idioma:
En
Ano de publicação:
2018
Tipo de documento:
Article