The landscape of mRNA nanomedicine

X Huang, N Kong, X Zhang, Y Cao, R Langer, W Tao - Nature Medicine, 2022 - nature.com
Messenger RNA (mRNA) is an emerging class of therapeutic agent for the prevention and
treatment of a wide range of diseases. The recent success of the two highly efficacious …

[PDF][PDF] Therapeutic in vivo delivery of gene editing agents

A Raguram, S Banskota, DR Liu - Cell, 2022 - cell.com
In vivo gene editing therapies offer the potential to treat the root causes of many genetic
diseases. Realizing the promise of therapeutic in vivo gene editing requires the ability to …

Chemical and biophysical signatures of the protein corona in nanomedicine

J Ren, N Andrikopoulos, K Velonia… - Journal of the …, 2022 - ACS Publications
An inconvenient hurdle in the practice of nanomedicine is the protein corona, a spontaneous
collection of biomolecular species by nanoparticles in living systems. The protein corona is …

Nanoparticle single-cell multiomic readouts reveal that cell heterogeneity influences lipid nanoparticle-mediated messenger RNA delivery

C Dobrowolski, K Paunovska… - Nature …, 2022 - nature.com
Cells that were previously described as homogeneous are composed of subsets with distinct
transcriptional states. However, it remains unclear whether this cell heterogeneity influences …

Efficient in vivo genome editing prevents hypertrophic cardiomyopathy in mice

D Reichart, GA Newby, H Wakimoto, M Lun… - Nature medicine, 2023 - nature.com
Dominant missense pathogenic variants in cardiac myosin heavy chain cause hypertrophic
cardiomyopathy (HCM), a currently incurable disorder that increases risk for stroke, heart …

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 …

Arginine supplementation targeting tumor-killing immune cells reconstructs the tumor microenvironment and enhances the antitumor immune response

J Zhang, S Wang, X Guo, Y Lu, X Liu, M Jiang, X Li… - ACS …, 2022 - ACS Publications
The tumor microenvironment (TME) is characterized by several immunosuppressive factors,
of which weak acidity and l-arginine (l-arg) deficiency are two common features. A weak …

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 …

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 …

A Unified Strategy to Improve Lipid Nanoparticle Mediated mRNA Delivery Using Adenosine Triphosphate

Y Ma, OS Fenton - Journal of the American Chemical Society, 2023 - ACS Publications
A central goal of chemical and drug delivery sciences is to maximize the therapeutic efficacy
of a given drug at the lowest possible dose. Here, we report a generalizable strategy that …