Drug delivery systems for CRISPR-based genome editors

V Madigan, F Zhang, JE Dahlman - Nature Reviews Drug Discovery, 2023 - nature.com
CRISPR-based drugs can theoretically manipulate any genetic target. In practice, however,
these drugs must enter the desired cell without eliciting an unwanted immune response, so …

The use of RNA-based treatments in the field of cancer immunotherapy

M Chehelgerdi, M Chehelgerdi - Molecular Cancer, 2023 - Springer
Over the past several decades, mRNA vaccines have evolved from a theoretical concept to a
clinical reality. These vaccines offer several advantages over traditional vaccine techniques …

Lipid nanoparticle-mediated lymph node–targeting delivery of mRNA cancer vaccine elicits robust CD8+ T cell response

J Chen, Z Ye, C Huang, M Qiu… - Proceedings of the …, 2022 - National Acad Sciences
The targeted delivery of messenger RNA (mRNA) to desired organs remains a great
challenge for in vivo applications of mRNA technology. For mRNA vaccines, the targeted …

Ligand-tethered lipid nanoparticles for targeted RNA delivery to treat liver fibrosis

X Han, N Gong, L Xue, MM Billingsley… - Nature …, 2023 - nature.com
Lipid nanoparticle-mediated RNA delivery holds great potential to treat various liver
diseases. However, targeted delivery of RNA therapeutics to activated liver-resident …

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 …

Engineered virus-like particles for transient delivery of prime editor ribonucleoprotein complexes in vivo

M An, A Raguram, SW Du, S Banskota, JR Davis… - Nature …, 2024 - nature.com
Prime editing enables precise installation of genomic substitutions, insertions and deletions
in living systems. Efficient in vitro and in vivo delivery of prime editing components, however …

Extrahepatic targeting of lipid nanoparticles in vivo with intracellular targeting for future nanomedicines

T Nakamura, Y Sato, Y Yamada… - Advanced drug delivery …, 2022 - Elsevier
A new era of nanomedicines that involve nucleic acids/gene therapy has been opened after
two decades in 21st century and new types of more efficient drug delivery systems (DDS) …

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 …

Phosphatidylserine lipid nanoparticles promote systemic RNA delivery to secondary lymphoid organs

S Luozhong, Z Yuan, T Sarmiento, Y Chen, W Gu… - Nano Letters, 2022 - ACS Publications
Secondary lymphoid organs (SLOs) are an important target for mRNA delivery in various
applications. While the current delivery method relies on the drainage of nanoparticles to …

Fast and facile synthesis of amidine-incorporated degradable lipids for versatile mRNA delivery in vivo

X Han, MG Alameh, N Gong, L Xue, M Ghattas… - Nature …, 2024 - nature.com
Lipid nanoparticles (LNPs) are widely used for mRNA delivery, with cationic lipids greatly
affecting biodistribution, cellular uptake, endosomal escape and transfection efficiency …