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 …

Recent advances in nanoparticulate RNA delivery systems

J Witten, Y Hu, R Langer, DG Anderson - Proceedings of the National …, 2024 - pnas.org
Nanoparticle-based RNA delivery has shown great progress in recent years with the
approval of two mRNA vaccines for Severe Acute Respiratory Syndrome Coronavirus 2 …

Combinatorial development of nebulized mRNA delivery formulations for the lungs

AY Jiang, J Witten, IO Raji, F Eweje, C MacIsaac… - Nature …, 2024 - nature.com
Inhaled delivery of mRNA has the potential to treat a wide variety of diseases. However,
nebulized mRNA lipid nanoparticles (LNPs) face several unique challenges including …

[HTML][HTML] Engineering nanoparticle toolkits for mRNA delivery

B Sun, W Wu, EA Narasipura, Y Ma, C Yu… - Advanced Drug Delivery …, 2023 - Elsevier
The concept of using mRNA to produce its own medicine in situ in the body makes it an ideal
drug candidate, holding great potential to revolutionize the way we approach medicine. The …

Physiological barriers and strategies of lipid‐based nanoparticles for nucleic acid drug delivery

M Hu, X Li, Z You, R Cai, C Chen - Advanced Materials, 2024 - Wiley Online Library
Lipid‐based nanoparticles (LBNPs) are currently the most promising vehicles for nucleic
acid drug (NAD) delivery. Although their clinical applications have achieved success, the …

AGILE platform: a deep learning powered approach to accelerate LNP development for mRNA delivery

Y Xu, S Ma, H Cui, J Chen, S Xu, F Gong… - Nature …, 2024 - nature.com
Ionizable lipid nanoparticles (LNPs) are seeing widespread use in mRNA delivery, notably
in SARS-CoV-2 mRNA vaccines. However, the expansion of mRNA therapies beyond …

Gene therapy for CNS disorders: modalities, delivery and translational challenges

J Gao, S Gunasekar, Z **a, K Shalin, C Jiang… - Nature Reviews …, 2024 - nature.com
Gene therapy is emerging as a powerful tool to modulate abnormal gene expression, a
hallmark of most CNS disorders. The transformative potentials of recently approved gene …

Frameworks for transformational breakthroughs in RNA-based medicines

JR Androsavich - Nature Reviews Drug Discovery, 2024 - nature.com
RNA has sparked a revolution in modern medicine, with the potential to transform the way
we treat diseases. Recent regulatory approvals, hundreds of new clinical trials, the …

Cellular trafficking of nanotechnology‐mediated mRNA delivery

P Huang, H Deng, C Wang, Y Zhou… - Advanced …, 2024 - Wiley Online Library
Messenger RNA (mRNA)‐based therapy has emerged as a powerful, safe, and rapidly
scalable therapeutic approach that involves technologies for both mRNA itself and the …

In situ combinatorial synthesis of degradable branched lipidoids for systemic delivery of mRNA therapeutics and gene editors

X Han, J Xu, Y Xu, MG Alameh, L Xue, N Gong… - Nature …, 2024 - nature.com
The ionizable lipidoid is a key component of lipid nanoparticles (LNPs). Degradable
lipidoids containing extended alkyl branches have received tremendous attention, yet their …