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- W4323804928 endingPage "487" @default.
- W4323804928 startingPage "471" @default.
- W4323804928 abstract "Most lung diseases are serious conditions resulting from genetic and environmental causes associated with high mortality and severe symptoms. Currently, treatments available have a palliative effect and many targets are still considered undruggable. Gene therapy stands as an attractive approach to offering innovative therapeutic solutions. CRISPRCas9 has established a remarkable potential for genome editing with high selectivity to targeted mutations. To ensure high efficacy with minimum systemic exposure, the delivery and administration route are key components that must be investigated.This review is focused on the delivery of CRISPRCas9 to the lungs, taking advantage of lipid nanoparticles (LNPs), the most clinically advanced nucleic acid carriers. We also aim to highlight the benefits of pulmonary administration as a local delivery route and the use of spray drying to prepare stable nucleic-acid-based dry powder formulations that can overcome multiple lung barriers.Exploring the pulmonary administration to deliver CRISPRCas9 loaded in LNPs as a dry powder increases the chances to achieve high efficacy and reduced adverse effects. CRISPRCas9 loaded in LNP-embedded microparticles has not yet been reported in the literature but has the potential to reach and accumulate in target cells in the lung, thus, enhancing overall efficacy and safety." @default.
- W4323804928 created "2023-03-11" @default.
- W4323804928 creator A5025193205 @default.
- W4323804928 creator A5035127057 @default.
- W4323804928 creator A5064069613 @default.
- W4323804928 creator A5065822348 @default.
- W4323804928 creator A5072006484 @default.
- W4323804928 date "2023-03-12" @default.
- W4323804928 modified "2023-10-05" @default.
- W4323804928 title "Shaping the future from the small scale: dry powder inhalation of CRISPR-Cas9 lipid nanoparticles for the treatment of lung diseases" @default.
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