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 …

Recent advances in siRNA delivery mediated by lipid-based nanoparticles

S Yonezawa, H Koide, T Asai - Advanced drug delivery reviews, 2020 - Elsevier
Small interfering RNA (siRNA) has been expected to be a unique pharmaceutic for the
treatment of broad-spectrum intractable diseases. However, its unfavorable properties such …

Chemically modified platforms for better RNA therapeutics

Y Shi, X Zhen, Y Zhang, Y Li, S Koo, Q Saiding… - Chemical …, 2024 - ACS Publications
RNA-based therapies have catalyzed a revolutionary transformation in the biomedical
landscape, offering unprecedented potential in disease prevention and treatment. However …

RNA interference in the era of nucleic acid therapeutics

V Jadhav, A Vaishnaw, K Fitzgerald, MA Maier - Nature Biotechnology, 2024 - nature.com
Two decades of research on RNA interference (RNAi) have transformed a breakthrough
discovery in biology into a robust platform for a new class of medicines that modulate mRNA …

Small interfering RNAs (siRNAs) in cancer therapy: a nano-based approach

G Mahmoodi Chalbatani, H Dana… - International journal …, 2019 - Taylor & Francis
Cancer is one of the most complex diseases that has resulted in multiple genetic disorders
and cellular abnormalities. Globally, cancer is the most common health concern disease that …

The medicinal chemistry of therapeutic oligonucleotides

WB Wan, PP Seth - Journal of medicinal chemistry, 2016 - ACS Publications
Oligonucleotide-based therapeutics have made rapid progress in the clinic for treatment of a
variety of disease indications. Unmodified oligonucleotides are polyanionic macromolecules …

[HTML][HTML] Effective tools for RNA-derived therapeutics: siRNA interference or miRNA mimicry

P Wang, Y Zhou, AM Richards - Theranostics, 2021 - ncbi.nlm.nih.gov
The approval of the first small interfering RNA (siRNA) drug Patisiran by FDA in 2018 marks
a new era of RNA interference (RNAi) therapeutics. MicroRNAs (miRNA), an important post …

Insight into the prospects for RNAi therapy of cancer

Z Tian, G Liang, K Cui, Y Liang, Q Wang, S Lv… - Frontiers in …, 2021 - frontiersin.org
RNA interference (RNAi), also known as gene silencing, is a biological process that
prevents gene expression in certain diseases such as cancer. It can be used to improve the …

Selection of GalNAc-conjugated siRNAs with limited off-target-driven rat hepatotoxicity

MM Janas, MK Schlegel, CE Harbison… - Nature …, 2018 - nature.com
Small interfering RNAs (siRNAs) conjugated to a trivalent N-acetylgalactosamine (GalNAc)
ligand are being evaluated in investigational clinical studies for a variety of indications. The …

Current prospects for RNA interference-based therapies

BL Davidson, PB McCray Jr - Nature Reviews Genetics, 2011 - nature.com
RNA interference (RNAi) is a powerful approach for reducing expression of endogenously
expressed proteins. It is widely used for biological applications and is being harnessed to …