Delivery of RNAs to specific organs by lipid nanoparticles for gene therapy

K Godbout, JP Tremblay - Pharmaceutics, 2022 - mdpi.com
Gene therapy holds great promise in the treatment of genetic diseases. It is now possible to
make DNA modifications using the CRISPR system. However, a major problem remains: the …

Ionizable drug delivery systems for efficient and selective gene therapy

YQ Zhang, RR Guo, YH Chen, TC Li, WZ Du… - Military Medical …, 2023 - Springer
Gene therapy has shown great potential to treat various diseases by repairing the abnormal
gene function. However, a great challenge in bringing the nucleic acid formulations to the …

Nanoparticle-based DNA vaccine protects against SARS-CoV-2 variants in female preclinical models

LC Guimaraes, PAC Costa, SRA Scalzo Júnior… - Nature …, 2024 - nature.com
A safe and effective vaccine with long-term protection against SARS-CoV-2 variants of
concern (VOCs) is a global health priority. Here, we develop lipid nanoparticles (LNPs) to …

Delivery of plasmid DNA by ionizable lipid nanoparticles to induce CAR expression in T cells

PHDM Prazeres, H Ferreira, PAC Costa… - International Journal …, 2023 - Taylor & Francis
Introduction Chimeric antigen receptor (CAR) cell therapy represents a hallmark in cancer
immunotherapy, with significant clinical results in the treatment of hematological tumors …

Structuring lipid nanoparticles, DNA, and protein corona into stealth bionanoarchitectures for in vivo gene delivery

S Renzi, L Digiacomo, D Pozzi, E Quagliarini… - Nature …, 2024 - nature.com
Lipid nanoparticles (LNPs) play a crucial role in addressing genetic disorders, and cancer,
and combating pandemics such as COVID-19 and its variants. Yet, the ability of LNPs to …

Gene editing innovations and their applications in cardiomyopathy research

E Kyriakopoulou, T Monnikhof… - Disease Models & …, 2023 - journals.biologists.com
Cardiomyopathies are among the major triggers of heart failure, but their clinical and genetic
complexity have hampered our understanding of these disorders and delayed the …

Optimized microfluidic formulation and organic excipients for improved lipid nanoparticle mediated genome editing

R Palanki, EL Han, AM Murray, R Maganti, S Tang… - Lab on a Chip, 2024 - pubs.rsc.org
mRNA-based gene editing platforms have tremendous promise in the treatment of genetic
diseases. However, for this potential to be realized in vivo, these nucleic acid cargos must …

[HTML][HTML] Navigating the landscape of RNA delivery systems in cardiovascular disease therapeutics

P Gil-Cabrerizo, T Simon-Yarza, E Garbayo… - Advanced Drug Delivery …, 2024 - Elsevier
Cardiovascular diseases (CVDs) stand as the leading cause of death worldwide, posing a
significant global health challenge. Consequently, the development of innovative …

[HTML][HTML] Evaluation of a DoE based approach for comprehensive modelling of the effect of lipid nanoparticle composition on nucleic acid delivery

Y Qin, AA Walters, N Rouatbi, JTW Wang… - Biomaterials, 2023 - Elsevier
Therapeutic nucleic acids (TNAs) comprise an alternative to conventional drugs for cancer
therapy. Recently, stable nucleic acid lipid particles (SNALPs) have been explored to deliver …

The menace of severe adverse events and deaths associated with viral gene therapy and its potential solution

A Kachanov, A Kostyusheva, S Brezgin… - Medicinal Research …, 2024 - Wiley Online Library
Over the past decade, in vivo gene replacement therapy has significantly advanced,
resulting in market approval of numerous therapeutics predominantly relying on adeno …