[HTML][HTML] Drug delivery systems for RNA therapeutics

K Paunovska, D Loughrey, JE Dahlman - Nature Reviews Genetics, 2022 - nature.com
RNA-based gene therapy requires therapeutic RNA to function inside target cells without
eliciting unwanted immune responses. RNA can be ferried into cells using non-viral drug …

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 …

Combinatorial design of nanoparticles for pulmonary mRNA delivery and genome editing

B Li, RS Manan, SQ Liang, A Gordon, A Jiang… - Nature …, 2023 - nature.com
The expanding applications of nonviral genomic medicines in the lung remain restricted by
delivery challenges. Here, leveraging a high-throughput platform, we synthesize and screen …

[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 …

Targeted drug delivery strategies for precision medicines

MT Manzari, Y Shamay, H Kiguchi, N Rosen… - Nature Reviews …, 2021 - nature.com
Progress in the field of precision medicine has changed the landscape of cancer therapy.
Precision medicine is propelled by technologies that enable molecular profiling, genomic …

Engineered biomaterials for in situ tissue regeneration

AK Gaharwar, I Singh, A Khademhosseini - Nature Reviews Materials, 2020 - nature.com
In situ tissue regeneration harnesses the body's regenerative potential to control cell
functions for tissue repair. The design of biomaterials for in situ tissue engineering requires …

Blood-brain barrier–penetrating single CRISPR-Cas9 nanocapsules for effective and safe glioblastoma gene therapy

Y Zou, X Sun, Q Yang, M Zheng, O Shimoni… - Science …, 2022 - science.org
We designed a unique nanocapsule for efficient single CRISPR-Cas9 capsuling,
noninvasive brain delivery and tumor cell targeting, demonstrating an effective and safe …

Throughput-scalable manufacturing of SARS-CoV-2 mRNA lipid nanoparticle vaccines

SJ Shepherd, X Han, AJ Mukalel… - Proceedings of the …, 2023 - National Acad Sciences
Lipid nanoparticles (LNPs) are a potent delivery technology that have made it possible for
the recent clinical breakthroughs in mRNA therapeutics and vaccines. A key challenge to the …

Selective organ targeting (SORT) nanoparticles for tissue-specific mRNA delivery and CRISPR–Cas gene editing

Q Cheng, T Wei, L Farbiak, LT Johnson… - Nature …, 2020 - nature.com
CRISPR–Cas gene editing and messenger RNA-based protein replacement therapy hold
tremendous potential to effectively treat disease-causing mutations with diverse cellular …

Microfluidic technologies and devices for lipid nanoparticle-based RNA delivery

M Maeki, S Uno, A Niwa, Y Okada, M Tokeshi - Journal of Controlled …, 2022 - Elsevier
In 2021, mRNA vaccines against COVID-19 were approved by the Food and Drug
Administration. mRNA vaccines are important for preventing severe COVID-19 and returning …