[Recent progress and prospect in oligonucleotide therapeutics].
Nihon Rinsho
; 73(6): 1057-65, 2015 Jun.
Article
em Ja
| MEDLINE
| ID: mdl-26065142
ABSTRACT
Remarkable progress has been made in chemical modification and nonviral delivery systems that improve the properties and efficacy of therapeutics oligonucleotides therapeutics, such as antisense oligonucleotide (ASO) and small interfering RNA(siRNA). ASOs act through various mechanisms including the degradation of mRNA by RNase H (gapmer-type ASO) and the modulation alternative splicing patterns(splice switching oligonucleotide). Recent favorable outcomes in clinical trials for cancers and genetic diseases such as familial amyloid polyneuropathy and Duchenne muscular dystrophy indicate high clinical potency of oligonucleotide therapeutics. Here we reviewed recent advances in basic properties and clinical applications of ASO and siRNA, and provide future perspective on oligonucleotide therapeutics.
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Base de dados:
MEDLINE
Assunto principal:
Pesquisa
/
RNA Mensageiro
/
Oligonucleotídeos Antissenso
/
RNA Interferente Pequeno
/
Neoplasias
Tipo de estudo:
Prognostic_studies
Limite:
Animals
/
Humans
Idioma:
Ja
Revista:
Nihon Rinsho
Ano de publicação:
2015
Tipo de documento:
Article