Noncoding RNA therapeutics—challenges and potential solutions

M Winkle, SM El-Daly, M Fabbri, GA Calin - Nature reviews Drug …, 2021 - nature.com
Therapeutic targeting of noncoding RNAs (ncRNAs), such as microRNAs (miRNAs) and
long noncoding RNAs (lncRNAs), represents an attractive approach for the treatment of …

Nano drug delivery systems for antisense oligonucleotides (ASO) therapeutics

T Ramasamy, HB Ruttala, S Munusamy… - Journal of Controlled …, 2022 - Elsevier
Cancer, infectious diseases, and metabolic and hereditary genetic disorders are a global
health burden affecting millions of people, with contemporary treatments offering limited …

[HTML][HTML] Deliver the promise: RNAs as a new class of molecular entities for therapy and vaccination

AM Yu, MJ Tu - Pharmacology & therapeutics, 2022 - Elsevier
The concepts of developing RNAs as new molecular entities for therapies have arisen again
and again since the discoveries of antisense RNAs, direct RNA-protein interactions …

Peptides as pharmacological carriers to the brain: promises, shortcomings and challenges

S Parrasia, I Szabò, M Zoratti… - Molecular …, 2022 - ACS Publications
Central nervous system (CNS) diseases are among the most difficult to treat, mainly
because the vast majority of the drugs fail to cross the blood-brain barrier (BBB) or to reach …

Self-amplified ROS production from fatty acid oxidation enhanced tumor immunotherapy by atorvastatin/PD-L1 siRNA lipopeptide nanoplexes

Y Gao, Z Song, L Jia, Y Tang, C Wang, X Zhao, H Hu… - Biomaterials, 2022 - Elsevier
Despite the important role of reactive oxygen species (ROS) in battling cancer, ROS
production with current approaches has been severely limited by the deficiency of oxy …

Strategies for improving the safety and RNAi efficacy of noncovalent peptide/siRNA nanocomplexes

J Wang, G Chen, N Liu, X Han, F Zhao, L Zhang… - Advances in Colloid and …, 2022 - Elsevier
In the past decades, the striking development of cationic polypeptides and cell-penetrating
peptides (CPPs) tailored for small interfering RNA (siRNA) delivery has been fuelled by the …

Nucleic acid-based therapeutics for the treatment of central nervous system disorders

R McCartan, O Khorkova, CH Volmar… - Frontiers in …, 2023 - frontiersin.org
Nucleic acid-based therapeutics (NBTs) are an emerging class of drugs with potential for the
treatment of a wide range of central nervous system conditions. To date, pertaining to CNS …

Therapeutic Oligonucleotides: An Outlook on Chemical Strategies to Improve Endosomal Trafficking

P Mangla, Q Vicentini, A Biscans - Cells, 2023 - mdpi.com
The potential of oligonucleotide therapeutics is undeniable as more than 15 drugs have
been approved to treat various diseases in the liver, central nervous system (CNS), and …

[HTML][HTML] Efficient systemic CNS delivery of a therapeutic antisense oligonucleotide with a blood-brain barrier-penetrating ApoE-derived peptide

YQ Yeoh, A Amin, B Cuic, D Tomas, BJ Turner… - Biomedicine & …, 2024 - Elsevier
Antisense oligonucleotide (ASO) has emerged as a promising therapeutic approach for
treating central nervous system (CNS) disorders by modulating gene expression with high …

Systemic administration of a novel Beclin 1-derived peptide significantly upregulates autophagy in the spinal motor neurons of autophagy reporter mice

A Amin, ND Perera, D Tomas, B Cuic… - International Journal of …, 2024 - Elsevier
Autophagy, an intracellular degradation system, plays a vital role in protecting cells by
clearing damaged organelles, pathogens, and protein aggregates. Autophagy upregulation …