Therapeutic efficacy in a hemophilia B model using a biosynthetic mRNA liver depot system [PDF]
DNA-based gene therapy has considerable therapeutic potential, but the challenges associated with delivery continue to limit progress. Messenger RNA (mRNA) has the potential to provide for transient production of therapeutic proteins, without the need ...
A Akinc +89 more
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The Delivery of mRNA Vaccines for Therapeutics
mRNA vaccines have been revolutionary in combating the COVID-19 pandemic in the past two years. They have also become a versatile tool for the prevention of infectious diseases and treatment of cancers.
Nitika, Jiao Wei, Ai-Min Hui
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In vitro and in vivo mRNA delivery using lipid-enveloped pHresponsive polymer nanoparticles [PDF]
Biodegradable core−shell structured nanoparticles with a poly(β-amino ester) (PBAE) core enveloped by a phospholipid bilayer shell were developed for in vivo mRNA delivery with a view toward delivery of mRNA-based vaccines.
Alexopoulou L. +91 more
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Nanomedicine-Based Approaches for mRNA Delivery [PDF]
Messenger RNA (mRNA) has immense potential for developing a wide range of therapies, including immunotherapy and protein replacement. As mRNA presents no risk of integration into the host genome and does not require nuclear entry for transfection, which allows protein production even in nondividing cells, mRNA-based approaches can be envisioned as safe
Uchida, Satoshi +3 more
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mRNA Delivery Platform Based on Bacterial Outer Membrane Vesicles for Tumor Vaccine
The rapid display and delivery method for customized tumor mRNA vaccines is limited. Herein, bacteria-derived outer membrane vesicles (OMVs) are employed as an mRNA delivery platform by surface engineering of an RNA-binding protein, L7Ae.
Xiaoyu Gao +3 more
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Viral delivery of antioxidant genes as a therapeutic strategy in experimental models of amyotrophic lateral sclerosis. [PDF]
Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disorder with no effective treatment to date. Despite its multi-factorial aetiology, oxidative stress is hypothesized to be one of the key pathogenic mechanisms.
Azzouz, M +6 more
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Optimization of Lipid Nanoformulations for Effective mRNA Delivery
Huiling Chen,1 Xuan Ren,2 Shi Xu,3 Dekui Zhang,4,* TiYun Han4,* 1Department of Hematology, Lanzhou University Second Hospital, Lanzhou, People’s Republic of China; 2Department of Endocrinology and Metabolism, Lanzhou University Second Hospital ...
Chen H, Ren X, Xu S, Zhang D, Han T
doaj
Messenger RNA (mRNA) has generated great attention due to its broad potential therapeutic applications, including vaccines, protein replacement therapy, and immunotherapy.
Xueliang Yu +9 more
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Oral, nasal and pharyngeal exposure to lipopolysaccharide causes a fetal inflammatory response in sheep. [PDF]
BackgroundA fetal inflammatory response (FIR) in sheep can be induced by intraamniotic or selective exposure of the fetal lung or gut to lipopolysaccharide (LPS).
Jobe, Alan H +7 more
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Insights into the kinetics of siRNA-mediated gene silencing from live-cell and live-animal bioluminescent imaging [PDF]
Small interfering RNA (siRNA) molecules are potent effectors of post-transcriptional gene silencing. Using noninvasive bioluminescent imaging and a mathematical model of siRNA delivery and function, the effects of target-specific and treatment-specific ...
Bartlett, Derek W., Davis, Mark E.
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