Clinical experiences with systemically administered siRNA-based therapeutics in cancer

JE Zuckerman, ME Davis - Nature reviews Drug discovery, 2015 - nature.com
Small interfering RNA (siRNA)-based therapies are emerging as a promising new
anticancer approach, and a small number of Phase I clinical trials involving patients with …

RNA interference-based therapy and its delivery systems

X Chen, LS Mangala, C Rodriguez-Aguayo… - Cancer and Metastasis …, 2018 - Springer
RNA interference (RNAi) is considered a highly specific approach for gene silencing and
holds tremendous potential for treatment of various pathologic conditions such as …

Differential fates of biomolecules delivered to target cells via extracellular vesicles

M Kanada, MH Bachmann, JW Hardy… - Proceedings of the …, 2015 - National Acad Sciences
Extracellular vesicles (EVs), specifically exosomes and microvesicles (MVs), are presumed
to play key roles in cell–cell communication via transfer of biomolecules between cells. The …

Augmented lipid-nanoparticle-mediated in vivo genome editing in the lungs and spleen by disrupting Cas9 activity in the liver

CD Sago, MP Lokugamage, D Loughrey… - Nature biomedical …, 2022 - nature.com
Systemically delivered lipid nanoparticles are preferentially taken up by hepatocytes. This
hinders the development of effective, non-viral means of editing genes in tissues other than …

Therapeutic potentials of gene silencing by RNA interference: principles, challenges, and new strategies

Y Deng, CC Wang, KW Choy, Q Du, J Chen, Q Wang… - Gene, 2014 - Elsevier
During recent decades there have been remarkable advances in biology, in which one of
the most important discoveries is RNA interference (RNAi). RNAi is a specific post …

Photoacoustic image-guided biomimetic nanoparticles targeting rheumatoid arthritis

J Chen, S Zeng, Q Xue, Y Hong, L Liu… - Proceedings of the …, 2022 - National Acad Sciences
The high level of reactive oxygen species (ROS) in the rheumatoid arthritis (RA)
microenvironment (RAM) and its persistent inflammatory nature can promote damage to …

Recent advances in targeted delivery of non-coding RNA-based therapeutics for atherosclerosis

X Li, H Qi, W Cui, Z Wang, X Fu, T Li, H Ma, Y Yang… - Molecular Therapy, 2022 - cell.com
Cardiovascular disease (CVD) has overtaken infectious illnesses as the leading cause of
mortality and disability worldwide. The pathology that underpins CVD is atherosclerosis …

[HTML][HTML] RNA interference as a novel treatment strategy for chronic hepatitis B infection

RWH Hui, LY Mak, WK Seto… - Clinical and molecular …, 2022 - ncbi.nlm.nih.gov
Chronic hepatitis B (CHB) is a major cause of liver-related morbidity and mortality.
Functional cure of CHB, defined as sustainable hepatitis B surface antigen (HBsAg) …

Progress toward in vivo use of siRNAs-II

GR Rettig, MA Behlke - Molecular therapy, 2012 - cell.com
RNA interference (RNAi) has been extensively employed for in vivo research since its use
was first demonstrated in mammalian cells 10 years ago. Design rules have improved, and it …

First-in-human mutation-targeted siRNA phase Ib trial of an inherited skin disorder

SA Leachman, RP Hickerson, ME Schwartz… - Molecular Therapy, 2010 - cell.com
The rare skin disorder pachyonychia congenita (PC) is an autosomal dominant syndrome
that includes a disabling plantar keratoderma for which no satisfactory treatment is currently …