TY - JOUR
T1 - Enhancing Cytoplasmic Expression of Exogenous mRNA Through Dynamic Mechanical Stimulation
AU - Chen, Jiawen
AU - Patel, Aneri
AU - Mir, Mohammad
AU - Hudock, Maria R.
AU - Pinezich, Meghan R.
AU - Guenthart, Brandon
AU - Bacchetta, Matthew
AU - Vunjak-Novakovic, Gordana
AU - Kim, Jinho
N1 - Publisher Copyright:
© 2024 Wiley-VCH GmbH.
PY - 2025/1/3
Y1 - 2025/1/3
N2 - Ionizable lipid nanoparticles (LNPs) are pivotal in combating COVID-19, and numerous preclinical and clinical studies have highlighted their potential in nucleic acid-based therapies and vaccines. However, the effectiveness of endosomal escape for the nucleic acid cargos encapsulated in LNPs is still low, leading to suboptimal treatment outcomes and side effects. Hence, improving endosomal escape is crucial for enhancing the efficacy of nucleic acid delivery using LNPs. Here, a mechanical oscillation (frequency: 65 Hz) is utilized to prompt the LNP-mediated endosomal escape. The results reveal this mechanical oscillation can induce the combination and fusion between LNPs with opposite surface charges, enhance endosomal escape of mRNA, and increase the transfection efficiency of mRNA. Additionally, cell viability remains high at 99.3% after treatment with oscillation, which is comparable to that of untreated cells. Furthermore, there is no obvious damage to mitochondrial membrane potential and Golgi apparatus integrity. Thus, this work presents a user-friendly and safe approach to enhancing endosomal escape of mRNA and boosting gene expression. As a result, this work can be potentially utilized in both research and clinical fields to facilitate LNP-based delivery by enabling more effective release of LNP-encapsulated cargos from endosomes.
AB - Ionizable lipid nanoparticles (LNPs) are pivotal in combating COVID-19, and numerous preclinical and clinical studies have highlighted their potential in nucleic acid-based therapies and vaccines. However, the effectiveness of endosomal escape for the nucleic acid cargos encapsulated in LNPs is still low, leading to suboptimal treatment outcomes and side effects. Hence, improving endosomal escape is crucial for enhancing the efficacy of nucleic acid delivery using LNPs. Here, a mechanical oscillation (frequency: 65 Hz) is utilized to prompt the LNP-mediated endosomal escape. The results reveal this mechanical oscillation can induce the combination and fusion between LNPs with opposite surface charges, enhance endosomal escape of mRNA, and increase the transfection efficiency of mRNA. Additionally, cell viability remains high at 99.3% after treatment with oscillation, which is comparable to that of untreated cells. Furthermore, there is no obvious damage to mitochondrial membrane potential and Golgi apparatus integrity. Thus, this work presents a user-friendly and safe approach to enhancing endosomal escape of mRNA and boosting gene expression. As a result, this work can be potentially utilized in both research and clinical fields to facilitate LNP-based delivery by enabling more effective release of LNP-encapsulated cargos from endosomes.
KW - endosomal escape
KW - gene expression
KW - ionizable lipid nanoparticles
KW - mechanical oscillation
KW - mRNA therapy
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U2 - 10.1002/adhm.202401918
DO - 10.1002/adhm.202401918
M3 - Article
C2 - 39440644
AN - SCOPUS:85207295059
SN - 2192-2640
VL - 14
JO - Advanced Healthcare Materials
JF - Advanced Healthcare Materials
IS - 1
M1 - 2401918
ER -