Unlocking the promise of mRNA therapeutics

E Rohner, R Yang, KS Foo, A Goedel… - Nature biotechnology, 2022 - nature.com
The extraordinary success of mRNA vaccines against coronavirus disease 2019 (COVID-19)
has renewed interest in mRNA as a means of delivering therapeutic proteins. Early clinical …

mRNA-based therapeutics: powerful and versatile tools to combat diseases

S Qin, X Tang, Y Chen, K Chen, N Fan… - Signal transduction and …, 2022 - nature.com
The therapeutic use of messenger RNA (mRNA) has fueled great hope to combat a wide
range of incurable diseases. Recent rapid advances in biotechnology and molecular …

[HTML][HTML] Lipid nanoparticles for mRNA delivery

X Hou, T Zaks, R Langer, Y Dong - Nature Reviews Materials, 2021 - nature.com
Messenger RNA (mRNA) has emerged as a new category of therapeutic agent to prevent
and treat various diseases. To function in vivo, mRNA requires safe, effective and stable …

Lipid nanoparticles (LNPs) for in vivo RNA delivery and their breakthrough technology for future applications

M Jeong, Y Lee, J Park, H Jung, H Lee - Advanced drug delivery reviews, 2023 - Elsevier
RNA therapeutics show a significant breakthrough for the treatment of otherwise incurable
diseases and genetic disorders by regulating disease-related gene expression. The …

mRNA nanomedicine: Design and recent applications

LJ Kubiatowicz, A Mohapatra, N Krishnan… - …, 2022 - Wiley Online Library
The rational design and application of mRNA‐based medicine have recently yielded some
key successes in the clinical management of human diseases. mRNA technology allows for …

Murine liver repair via transient activation of regenerative pathways in hepatocytes using lipid nanoparticle-complexed nucleoside-modified mRNA

F Rizvi, E Everton, AR Smith, H Liu, E Osota… - Nature …, 2021 - nature.com
Induction of intrinsic liver regeneration is an unmet need that can be achieved by temporally
activating key hepatocyte regenerative pathways. Here, we establish an efficient, safe, non …

Non-immunotherapy application of LNP-mRNA: Maximizing efficacy and safety

I Vlatkovic - Biomedicines, 2021 - mdpi.com
Lipid nanoparticle (LNP) formulated messenger RNA-based (LNP-mRNA) vaccines came
into the spotlight as the first vaccines against SARS-CoV-2 virus to be applied worldwide …

Lipid nanoparticle delivery systems to enable mRNA-based therapeutics

SC Semple, R Leone, CJ Barbosa, YK Tam, PJC Lin - Pharmaceutics, 2022 - mdpi.com
The world raced to develop vaccines to protect against the rapid spread of SARS-CoV-2
infection upon the recognition of COVID-19 as a global pandemic. A broad spectrum of …

Self-amplifying RNA approach for protein replacement therapy

D Papukashvili, N Rcheulishvili, C Liu, Y Ji… - International journal of …, 2022 - mdpi.com
Messenger RNA (mRNA) technology has already been successfully tested preclinically and
there are ongoing clinical trials for protein replacement purposes; however, more effort has …

mRNA therapy restores euglycemia and prevents liver tumors in murine model of glycogen storage disease

J Cao, M Choi, E Guadagnin, M Soty, M Silva… - Nature …, 2021 - nature.com
Abstract Glycogen Storage Disease 1a (GSD1a) is a rare, inherited metabolic disorder
caused by deficiency of glucose 6-phosphatase (G6Pase-α). G6Pase-α is critical for …