[HTML][HTML] Emerging concepts of miRNA therapeutics: from cells to clinic

C Diener, A Keller, E Meese - Trends in Genetics, 2022 - cell.com
MicroRNAs (miRNAs) are very powerful genetic regulators, as evidenced by the fact that a
single miRNA can direct entire cellular pathways via interacting with a broad spectrum of …

Advances of nanoparticles as drug delivery systems for disease diagnosis and treatment

R Liu, C Luo, Z Pang, J Zhang, S Ruan, M Wu… - Chinese chemical …, 2023 - Elsevier
Decades have passed since the first nanoparticles-base medicine was approved for human
cancer treatment, and the research and development of nanoparticles for drug delivery are …

[HTML][HTML] Therapeutic siRNA: state of the art

B Hu, L Zhong, Y Weng, L Peng, Y Huang… - Signal transduction and …, 2020 - nature.com
ABSTRACT RNA interference (RNAi) is an ancient biological mechanism used to defend
against external invasion. It theoretically can silence any disease-related genes in a …

Glycoconjugates: synthesis, functional studies, and therapeutic developments

SS Shivatare, VS Shivatare, CH Wong - Chemical reviews, 2022 - ACS Publications
Glycoconjugates are major constituents of mammalian cells that are formed via covalent
conjugation of carbohydrates to other biomolecules like proteins and lipids and often …

LYTACs that engage the asialoglycoprotein receptor for targeted protein degradation

G Ahn, SM Banik, CL Miller, NM Riley… - Nature Chemical …, 2021 - nature.com
Selective protein degradation platforms have afforded new development opportunities for
therapeutics and tools for biological inquiry. The first lysosome-targeting chimeras (LYTACs) …

Chemistry, mechanism and clinical status of antisense oligonucleotides and duplex RNAs

X Shen, DR Corey - Nucleic acids research, 2018 - academic.oup.com
RNA plays a central role in the expression of all genes. Because any sequence within RNA
can be recognized by complementary base pairing, synthetic oligonucleotides and …

GalNAc-siRNA conjugates: leading the way for delivery of RNAi therapeutics

AD Springer, SF Dowdy - Nucleic acid therapeutics, 2018 - liebertpub.com
Short-interfering RNA (siRNA)-induced RNAi responses have great potential to treat a wide
variety of human diseases from cancer to pandemic viral outbreaks to Parkinson's Disease …

[HTML][HTML] Antisense oligonucleotides: an emerging area in drug discovery and development

K Dhuri, C Bechtold, E Quijano, H Pham… - Journal of clinical …, 2020 - mdpi.com
Antisense oligonucleotides (ASOs) bind sequence specifically to the target RNA and
modulate protein expression through several different mechanisms. The ASO field is an …

[HTML][HTML] Delivery of oligonucleotides to the liver with GalNAc: from research to registered therapeutic drug

AJ Debacker, J Voutila, M Catley, D Blakey, N Habib - Molecular Therapy, 2020 - cell.com
Targeted delivery of oligonucleotides to liver hepatocytes using N-acetylgalactosamine
(GalNAc) conjugates that bind to the asialoglycoprotein receptor has become a …

Rekindling RNAi therapy: materials design requirements for in vivo siRNA delivery

B Kim, JH Park, MJ Sailor - Advanced materials, 2019 - Wiley Online Library
With the recent FDA approval of the first siRNA‐derived therapeutic, RNA interference
(RNAi)‐mediated gene therapy is undergoing a transition from research to the clinical …