Strategies, design, and chemistry in siRNA delivery systems

Y Dong, DJ Siegwart, DG Anderson - Advanced drug delivery reviews, 2019 - Elsevier
Emerging therapeutics that utilize RNA interference (RNAi) have the potential to treat broad
classes of diseases due to their ability to reversibly silence target genes. In August 2018, the …

Degradable controlled-release polymers and polymeric nanoparticles: mechanisms of controlling drug release

N Kamaly, B Yameen, J Wu, OC Farokhzad - Chemical reviews, 2016 - ACS Publications
The development of biodegradable polymers represents a revolution in medicine spanning
over 50 years and leading to significant biotechnological advancements in drug delivery …

Optimization of lipid nanoparticles for the delivery of nebulized therapeutic mRNA to the lungs

MP Lokugamage, D Vanover, J Beyersdorf… - Nature biomedical …, 2021 - nature.com
Lipid nanoparticles (LNPs) for the efficient delivery of drugs need to be designed for the
particular administration route and type of drug. Here we report the design of LNPs for the …

Piperazine-derived lipid nanoparticles deliver mRNA to immune cells in vivo

H Ni, MZC Hatit, K Zhao, D Loughrey… - Nature …, 2022 - nature.com
In humans, lipid nanoparticles (LNPs) have safely delivered therapeutic RNA to hepatocytes
after systemic administration and to antigen-presenting cells after intramuscular injection …

Zwitterionic phospholipidation of cationic polymers facilitates systemic mRNA delivery to spleen and lymph nodes

S Liu, X Wang, X Yu, Q Cheng… - Journal of the …, 2021 - ACS Publications
Polymers represent a promising therapeutic platform for extrahepatic messenger RNA
(mRNA) delivery but are hampered by low in vivo efficacy due to polyplex serum instability …

Nanogels: An overview of properties, biomedical applications and obstacles to clinical translation

KS Soni, SS Desale, TK Bronich - Journal of Controlled Release, 2016 - Elsevier
Nanogels have emerged as a versatile hydrophilic platform for encapsulation of guest
molecules with a capability to respond to external stimuli that can be used for a multitude of …

Targeted polymeric therapeutic nanoparticles: design, development and clinical translation

N Kamaly, Z **ao, PM Valencia… - Chemical Society …, 2012 - pubs.rsc.org
Polymeric materials have been used in a range of pharmaceutical and biotechnology
products for more than 40 years. These materials have evolved from their earlier use as …

Core-crosslinked polymeric micelles: Principles, preparation, biomedical applications and clinical translation

M Talelli, M Barz, CJF Rijcken, F Kiessling… - Nano today, 2015 - Elsevier
Polymeric micelles (PM) are extensively used to improve the delivery of hydrophobic drugs.
Many different PM have been designed and evaluated over the years, and some of them …

Degradable lipid nanoparticles with predictable in vivo siRNA delivery activity

KA Whitehead, JR Dorkin, AJ Vegas, PH Chang… - Nature …, 2014 - nature.com
One of the most significant challenges in the development of clinically viable delivery
systems for RNA interference therapeutics is to understand how molecular structures …

Lipid nanoparticle-delivered chemically modified mRNA restores chloride secretion in cystic fibrosis

E Robinson, KD MacDonald, K Slaughter, M McKinney… - Molecular Therapy, 2018 - cell.com
The promise of gene therapy for the treatment of cystic fibrosis has yet to be fully clinically
realized despite years of effort toward correcting the underlying genetic defect in the cystic …