Receptor-Mediated Uptake of Phosphorothioate Antisense Oligonucleotides in Different Cell Types of the Liver.
Nucleic Acid Ther
; 28(3): 119-127, 2018 06.
Article
en En
| MEDLINE
| ID: mdl-29425080
ABSTRACT
Oligonucleotide therapeutics have emerged as a third distinct platform for drug discovery within the pharmaceutical industry. Five oligonucleotide-based drugs have been approved by the US FDA and over 100 oligonucleotides drugs are currently at different stages of human trials. Several of these oligonucleotide drugs are modified using the phosphorothioate (PS) backbone modification where one of the nonbridging oxygen atoms of the phosphodiester linkage is replaced with sulfur. In this review, we summarize our knowledge on receptor-mediated uptake of PS antisense oligonucleotides (ASOs) within different cell types of the liver-a privileged organ for the discovery of oligonucleotide-based therapeutics.
Palabras clave
Texto completo:
1
Colección:
01-internacional
Base de datos:
MEDLINE
Asunto principal:
Glicoconjugados
/
Oligonucleótidos Antisentido
/
Técnicas de Transferencia de Gen
/
ARN Interferente Pequeño
/
Endocitosis
/
Oligonucleótidos Fosforotioatos
Límite:
Animals
/
Humans
Idioma:
En
Revista:
Nucleic Acid Ther
Año:
2018
Tipo del documento:
Article