Engineering precision nanoparticles for drug delivery

MJ Mitchell, MM Billingsley, RM Haley… - Nature reviews drug …, 2021 - nature.com
In recent years, the development of nanoparticles has expanded into a broad range of
clinical applications. Nanoparticles have been developed to overcome the limitations of free …

Future therapies for cystic fibrosis

L Allen, L Allen, SB Carr, G Davies, D Downey… - Nature …, 2023 - nature.com
We are currently witnessing transformative change for people with cystic fibrosis with the
introduction of small molecule, mutation-specific drugs capable of restoring function of the …

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 …

Strategies for non-viral vectors targeting organs beyond the liver

J Kim, Y Eygeris, RC Ryals, A Jozić, G Sahay - Nature nanotechnology, 2024 - nature.com
In recent years, nanoparticles have evolved to a clinical modality to deliver diverse nucleic
acids. Rising interest in nanomedicines comes from proven safety and efficacy profiles …

[HTML][HTML] Strategies to overcome the main challenges of the use of CRISPR/Cas9 as a replacement for cancer therapy

MF Rasul, BM Hussen, A Salihi, BS Ismael, PJ Jalal… - Molecular Cancer, 2022 - Springer
CRISPR/Cas9 (clustered regularly interspaced short palindromic repeats-associated protein
9) shows the opportunity to treat a diverse array of untreated various genetic and …

The delivery challenge: fulfilling the promise of therapeutic genome editing

J van Haasteren, J Li, OJ Scheideler, N Murthy… - Nature …, 2020 - nature.com
Genome editing has the potential to treat an extensive range of incurable monogenic and
complex diseases. In particular, advances in sequence-specific nuclease technologies have …

Gene therapy clinical trials worldwide to 2017: An update

SL Ginn, AK Amaya, IE Alexander… - The journal of gene …, 2018 - Wiley Online Library
To date, almost 2600 gene therapy clinical trials have been completed, are ongoing or have
been approved worldwide. Our database brings together global information on gene therapy …

Inhaled nanoformulated mRNA polyplexes for protein production in lung epithelium

AK Patel, JC Kaczmarek, S Bose… - Advanced …, 2019 - Wiley Online Library
Noninvasive aerosol inhalation is an established method of drug delivery to the lung, and
remains a desirable route for nucleic‐acid‐based therapeutics. In vitro transcribed (IVT) …

Lipid-based DNA therapeutics: hallmarks of non-viral gene delivery

J Buck, P Grossen, PR Cullis, J Huwyler… - ACS …, 2019 - ACS Publications
Gene therapy is a promising strategy for the treatment of monogenic disorders. Non-viral
gene delivery systems including lipid-based DNA therapeutics offer the opportunity to …

Progress in therapies for cystic fibrosis

K De Boeck, MD Amaral - The Lancet Respiratory Medicine, 2016 - thelancet.com
Standard follow-up and symptomatic treatment have allowed most patients with cystic
fibrosis to live to young adulthood. However, many patients still die prematurely from …