Results 21 to 30 of about 797,089 (324)

mRNA in the Context of Protein Replacement Therapy

Pharmaceutics, 2023
Protein replacement therapy is an umbrella term used for medical treatments that aim to substitute or replenish specific protein deficiencies that result either from the protein being absent or non-functional due to mutations in affected patients ...
Theofanis Vavilis, Eleni Stamoula, Alexandra Ainatzoglou, Athanasios Sachinidis, Malamatenia Lamprinou, Ioannis Dardalas, Ioannis S. Vizirianakis   +6 more
doaj   +4 more sources

Enhancing mRNA therapy through iterative delivery. [PDF]

Sci Adv
Clinical translation of therapeutic messenger RNA (mRNA) technologies, particularly in solid tumors, has been limited due to unavailability of effective delivery systems. Here, we describe an mRNA delivery system that overcomes current challenges by co-encapsulating gold nanoparticles (AuNPs) and mRNA within non-ionic surfactant vehicles (NSVs) to ...
Seaberg J, Gulla SK, Labombarde J, Roy R, Dwivedi SKD, Xu C, Kovats S, Bhattacharya R, Mukherjee P.   +8 more
europepmc   +3 more sources

Targeted mRNA Therapy for Ornithine Transcarbamylase Deficiency. [PDF]

Mol Ther, 2018
We describe a novel, two-nanoparticle mRNA delivery system and show that it is highly effective as a means of intracellular enzyme replacement therapy (i-ERT) using a murine model of ornithine transcarbamylase deficiency (OTCD). Our Hybrid mRNA Technology delivery system (HMT) comprises an inert lipid nanoparticle that protects the mRNA from nucleases ...
Prieve MG, Harvie P, Monahan SD, Roy D, Li AG, Blevins TL, Paschal AE, Waldheim M, Bell EC, Galperin A, Ella-Menye JR, Houston ME.   +11 more
europepmc   +4 more sources

Pre-mRNA splicing-associated diseases and therapies

RNA Biology, 2023
Precursor mRNA (pre-mRNA) splicing is an essential step in human gene expression and is carried out by a large macromolecular machine called the spliceosome. Given the spliceosome's role in shaping the cellular transcriptome, it is not surprising that mutations in the splicing machinery can result in a range of human diseases and disorders ...
Sierra L. Love, Joseph D. Emerson, Kazunori Koide, Aaron A. Hoskins   +3 more
openaire   +3 more sources

Superpulsed low-level laser therapy protects skeletal muscle of mdx mice against damage, inflammation and morphological changes delaying dystrophy progression. [PDF]

, 2014
Aim: To evaluate the effects of preventive treatment with low-level laser therapy (LLLT) on progression of dystrophy in mdx mice. Methods: Ten animals were randomly divided into 2 experimental groups treated with superpulsed LLLT (904 nm, 15 mW, 700 Hz ...
A Gur, A Stergioulas, AP Gautam, B Hegedus, BJ Barber, CF Rizzi, CR Hayworth, D Avni, DE Lebel, EC Leal Junior, EC Leal Junior, EC Leal Junior, EC Leal Junior, EC Leal Junior, EC Leal Junior, EC Leal Junior, Ernesto Cesar Pinto Leal-Junior, F Aimbire, F Aimbire, F Serra, GD Thomas, GF Cox, JA Goldman, Jan Magnus Bjordal, JK McGeachie, JM Bjordal, Jon Joensen, JR Basford, JS Chamberlain, KS Ramaswamy, Lucio Frigo, M Koenig, M Li, M Tullberg, Mark I. Johnson, Maurilio Sampaolesi, MM Schubert, O Ozcelik, P de Almeida, P de Almeida, Patrícia de Almeida, Paulo de Tarso Camillo de Carvalho, PC Silveira, R Albertini, R Albertini, RJ Fairclough, Rodrigo Álvaro Brandão Lopes-Martins, RT Chow, RT Chow, S Rao, S Rochkind, Shaiane Silva Tomazoni, T De Marchi, T Takema, TA Partridge, V Ricotti, W Lim, X Xu, Y Lampl, Y Moriyama, YY Huang   +60 more
core   +8 more sources

Inhibition of Murine Cytomegalovirus Infection in Animals by RNase P-Associated External Guide Sequences. [PDF]

, 2017
External guide sequence (EGS) RNAs are associated with ribonuclease P (RNase P), a tRNA processing enzyme, and represent promising agents for gene-targeting applications as they can direct RNase-P-mediated cleavage of a target mRNA.
Li, Wei, LIU, Fenyong, Liu, Yujun, Lu, Sangwei, Sheng, Jingxue, Sun, Xu, Trang, Phong, Vu, Gia-Phong, Xu, Mengqiong, Yang, Zhu   +9 more
core   +7 more sources

Preconditioning of mesenchymal stromal cells with low-intensity ultrasound: influence on chondrogenesis and directed SOX9 signaling pathways [PDF]

, 2020
Background: Continuous low-intensity ultrasound (cLIUS) facilitates the chondrogenic differentiation of human mesenchymal stromal cells (MSCs) in the absence of exogenously added transforming growth factor-beta (TGFβ) by upregulating the expression of ...
A Argentaro, A Augello, A Katsumi, A Woods, A Woods, AD Miller, AH Huang, B Yang, BH Choi, C Huang, C Matta, CA Simmons, CB Little, CJ O’Conor, CY Huang, D Schumann, DR Haudenschild, EM Darling, F Dell'Accio, G Coricor, H Sim, H-C Hsu, HJ Kwon, HJ Lee, HJ Lee, J Li, J Puetzer, JC Sng, JD Kisiday, JH Cui, JH Cui, JW Choi, K Ebisawa, K Pelttari, L Bian, Li Ling, LJ Ng, M Shakibaei, M Tsuda, MA Corrigan, MB Mueller, N Fahy, N Thelin, NP Whitney, P Müller, R Ogawa, RL Mauck, RM Delaine-Smith, RS Tigli, S Murakami, S Noriega, S Noriega, S Yamaguchi, SD Cook, SD Cook, T Furumatsu, T Guo, T Ito, T Juhasz, T Nishida, T Sanjukta Guha, TM Louw, V Lefebvre, V Lefebvre, W Huang, W Huang, Z Li, ZJ Zhang   +67 more
core   +2 more sources

mRNA: Fulfilling the Promise of Gene Therapy [PDF]

Molecular Therapy, 2015
In vitro–transcribed mRNA (IVT mRNA) is emerging as a new class of drug that has the potential to play a role in gene therapy that once was envisioned for DNA.1 Although first described as a therapeutic in 1992,2 IVT mRNA’s immunogenicity prevented its development for protein replacement therapies.
Weissman, Drew, Karikó, Katalin
openaire   +2 more sources

Lipid Nanoparticles for Organ-Specific mRNA Therapeutic Delivery

Pharmaceutics, 2021
Advances in the using in vitro transcribed (IVT) modRNA in the past two decades, especially the tremendous recent success of mRNA vaccines against SARS-CoV-2, have brought increased attention to IVT mRNA technology.
Magdalena M. Żak, Lior Zangi
doaj   +1 more source

Ionizable Lipid Nanoparticles for mRNA Delivery

Advanced NanoBiomed Research, 2023
Therapeutics based on messenger RNA (mRNA) have become a promising class of methods for various diseases’ treatments in recent years. The safe and effective delivery of mRNA into target cells is the premise of the clinical translation of mRNA. Due to the
Xuefeng Tang, Ying Zhang, Xiaojun Han
doaj   +1 more source