RNA modified with acyclic threoninol nucleic acids for RNA interference

Upon the discovery of the RNA interference pathway, the development of nucleic acids derivatives for therapeutic purposes has soon caught the attention of biomedical researchers. Although synthetic small interfering RNA (siRNA) has been extensively used to downregulate any protein-coding mRNA, sever...

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Detalles Bibliográficos
Autores: Alagia, Adele, Terrazas, Montserrat, Eritja Casadellà, Ramón
Tipo de recurso: artículo
Estado:Versión publicada
Fecha de publicación:2015
País:España
Institución:Consejo Superior de Investigaciones Científicas (CSIC)
Repositorio:DIGITAL.CSIC. Repositorio Institucional del CSIC
OAI Identifier:oai:digital.csic.es:10261/126536
Acceso en línea:http://hdl.handle.net/10261/126536
Access Level:acceso abierto
Palabra clave:RNA interference
sIRNAS
acyclic nucleic acids
Descripción
Sumario:Upon the discovery of the RNA interference pathway, the development of nucleic acids derivatives for therapeutic purposes has soon caught the attention of biomedical researchers. Although synthetic small interfering RNA (siRNA) has been extensively used to downregulate any protein-coding mRNA, several key issues still remain unsolved. The acyclic threoninol nucleic acid (aTNA), placed at certain siRNA positions, is a useful modification to reduce the oligonucleotides vulnerability towards nucleases. In addition, it can be exploited to avoid several OFF-target effects that limit the biological safety of the RNAi-based agents.