Bioconjugates for improved delivery of oligonucleotide therapeutics to the central nervous system
Chungbuk National University · University of Michigan · +1 more institution
Abstract
Oligonucleotide therapeutics, including antisense oligonucleotides (ASOs) and small interfering RNAs (siRNAs), have gained increasing attention as a novel modality for gene-targeted interventions for central nervous system (CNS) disorders, particularly in the context of rare and inherited neurological conditions. By correcting pathogenic abnormalities in gene splicing or expression, oligonucleotide therapeutics offer a combination of extreme specificity and disease-modifying or even curative effects. However, achieving robust delivery to the CNS after systemic administration remains a significant challenge due to the presence of the blood-brain barrier and the intrinsic physicochemical limitations of…
Citation impact
- FWCI
- 36.03
- Percentile
- 99%
- References
- 181
Authors
4Topics & keywords
- Oligonucleotide
- Central nervous system
- Context (archaeology)
- Drug delivery
- RNA splicing
- Delivery system