scholarly journals Erratum: Spontaneous cellular uptake of exogenous messenger RNA in vivo is nucleic acid-specific, saturable and ion dependent

Gene Therapy ◽  
2009 ◽  
Vol 16 (5) ◽  
pp. 706-706 ◽  
Author(s):  
J Probst ◽  
B Weide ◽  
B Scheel ◽  
B J Pichler ◽  
I Hoerr ◽  
...  
Gene Therapy ◽  
2007 ◽  
Vol 14 (15) ◽  
pp. 1175-1180 ◽  
Author(s):  
J Probst ◽  
B Weide ◽  
B Scheel ◽  
B J Pichler ◽  
I Hoerr ◽  
...  

2012 ◽  
Vol 7 (7) ◽  
pp. 1276-1280 ◽  
Author(s):  
Mateo I. Sánchez ◽  
José Martínez-Costas ◽  
Francisco Gonzalez ◽  
María A. Bermudez ◽  
M. Eugenio Vázquez ◽  
...  

1972 ◽  
Vol 71 (2_Suppla) ◽  
pp. S369-S380 ◽  
Author(s):  
Francis T. Kenney ◽  
Kai-Lin Lee ◽  
Charles D. Stiles

ABSTRACT Analyses of the response of hydrocortisone-induced tyrosine transaminase in cultured H-35 cells to inhibitors of translation (cycloheximide, puromycin) suggest: (1) that bound ribosomes stabilize messenger RNA in vivo; (2) that messenger is degraded at a rate determined by the rate of translation. Since specific messenger RNAs of mammalian cells are degraded at quite different rates, there may be extensive heterogeneity either in the rate at which ribosomes traverse different messengers or in the number of ribosomes which translate specific messenger RNAs.


ACS Nano ◽  
2021 ◽  
Author(s):  
Tyler D. Gavitt ◽  
Alyssa K. Hartmann ◽  
Shraddha S. Sawant ◽  
Arlind B. Mara ◽  
Steven M. Szczepanek ◽  
...  
Keyword(s):  

2021 ◽  
Vol 7 (9) ◽  
pp. eabf4398
Author(s):  
M. Kim ◽  
M. Jeong ◽  
S. Hur ◽  
Y. Cho ◽  
J. Park ◽  
...  

Ionizable lipid nanoparticles (LNPs) have been widely used for in vivo delivery of RNA therapeutics into the liver. However, a main challenge remains to develop LNP formulations for selective delivery of RNA into certain types of liver cells, such as hepatocytes and liver sinusoidal endothelial cells (LSECs). Here, we report the engineered LNPs for the targeted delivery of RNA into hepatocytes and LSECs. The effects of particle size and polyethylene glycol–lipid content in the LNPs were evaluated for the hepatocyte-specific delivery of mRNA by ApoE-mediated cellular uptake through low-density lipoprotein receptors. Targeted delivery of RNA to LSECs was further investigated using active ligands. Incorporation of mannose allowed the selective delivery of RNA to LSECs, while minimizing the unwanted cellular uptake by hepatocytes. These results demonstrate that engineered LNPs have great potential for the cell type–specific delivery of RNA into the liver and other tissues.


Cells ◽  
2021 ◽  
Vol 10 (5) ◽  
pp. 1011
Author(s):  
Karishma Dhuri ◽  
Rutesh N. Vyas ◽  
Leslie Blumenfeld ◽  
Rajkumar Verma ◽  
Raman Bahal

Ischemic stroke and factors modifying ischemic stroke responses, such as social isolation, contribute to long-term disability worldwide. Several studies demonstrated that the aberrant levels of microRNAs contribute to ischemic stroke injury. In prior studies, we established that miR-141-3p increases after ischemic stroke and post-stroke isolation. Herein, we explored two different anti-miR oligonucleotides; peptide nucleic acid (PNAs) and phosphorothioates (PS) for ischemic stroke therapy. We used US FDA approved biocompatible poly (lactic-co-glycolic acid) (PLGA)-based nanoparticle formulations for delivery. The PNA and PS anti-miRs were encapsulated in PLGA nanoparticles by double emulsion solvent evaporation technique. All the formulated nanoparticles showed uniform morphology, size, distribution, and surface charge density. Nanoparticles also exhibited a controlled nucleic acid release profile for 48 h. Further, we performed in vivo studies in the mouse model of ischemic stroke. Ischemic stroke was induced by transient (60 min) occlusion of middle cerebral artery occlusion followed by a reperfusion for 48 or 72 h. We assessed the blood-brain barrier permeability of PLGA NPs containing fluorophore (TAMRA) anti-miR probe after systemic delivery. Confocal imaging shows uptake of fluorophore tagged anti-miR in the brain parenchyma. Next, we evaluated the therapeutic efficacy after systemic delivery of nanoparticles containing PNA and PS anti-miR-141-3p in mice after stroke. Post-treatment differentially reduced both miR-141-3p levels in brain tissue and infarct injury. We noted PNA-based anti-miR showed superior efficacy compared to PS-based anti-miR. Herein, we successfully established that nanoparticles encapsulating PNA or PS-based anti-miRs-141-3p probes could be used as a potential treatment for ischemic stroke.


Pharmaceutics ◽  
2020 ◽  
Vol 13 (1) ◽  
pp. 45
Author(s):  
Iman M. Alfagih ◽  
Basmah Aldosari ◽  
Bushra AlQuadeib ◽  
Alanood Almurshedi ◽  
Mariyam M. Alfagih

Messenger RNA (mRNA)-based vaccines have shown promise against infectious diseases and several types of cancer in the last two decades. Their promise can be attributed to their safety profiles, high potency, and ability to be rapidly and affordably manufactured. Now, many RNA-based vaccines are being evaluated in clinical trials as prophylactic and therapeutic vaccines. However, until recently, their development has been limited by their instability and inefficient in vivo transfection. The nanodelivery system plays a dual function in RNA-based vaccination by acting as a carrier system and as an adjuvant. That is due to its similarity to microorganisms structurally and size-wise; the nanodelivery system can augment the response by the immune system via simulating the natural infection process. Nanodelivery systems allow non-invasive mucosal administration, targeted immune cell delivery, and controlled delivery, reducing the need for multiple administrations. They also allow co-encapsulating with immunostimulators to improve the overall adjuvant capacity. The aim of this review is to discuss the recent developments and applications of biodegradable nanodelivery systems that improve RNA-based vaccine delivery and enhance the immunological response against targeted diseases.


1986 ◽  
Vol 59 (6) ◽  
pp. 679-695 ◽  
Author(s):  
Jeffrey L. Price ◽  
Brian B. Gourlie ◽  
Yuan Lin ◽  
Ru Chih C. Huang

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