Molecular Therapy: Oncolytics, 2022 Glioblastoma (GBM) is characterized as having high molecular heterogeneity and complexity, which can be well revealed by genomic study. A truly effective treatment for GBM should flexibly address its heterogeneities, complexity, and strong drug ...
Xiangjun Tang +13 more
doaj +4 more sources
A lipid nanoparticle-based oligodendrocyte-specific mRNA therapy [PDF]
Molecular Therapy: Nucleic AcidsDespite the wide range of applications of mRNA therapies, major difficulties exist in the efficient delivery of mRNA into oligodendrocytes, a type of glial cell in the brain. Commonly used viral vectors are not efficient in transforming oligodendrocytes.
Masanori Sawamura +13 more
doaj +4 more sources
Development of an mRNA replacement therapy for phenylketonuria
Molecular Therapy: Nucleic Acids, 2022 Phenylketonuria (PKU) is an inborn error caused by deficiencies in phenylalanine (Phe) metabolism. Mutations in the phenylalanine hydroxylase (PAH) gene are the main cause of the disease whose signature hallmarks of toxically elevated levels of Phe ...
Carlos G. Perez-Garcia +11 more
doaj +4 more sources
mRNA-Enhanced Cell Therapy and Cardiovascular Regeneration [PDF]
Cells, 2021 mRNA has emerged as an important biomolecule in the global call for the development of therapies during the COVID-19 pandemic. Synthetic in vitro-transcribed (IVT) mRNA can be engineered to mimic naturally occurring mRNA and can be used as a tool to ...
Palas K. Chanda +2 more
doaj +3 more sources
Lipid-mRNA nanoparticles landscape for cancer therapy
Frontiers in Bioengineering and Biotechnology, 2022 Intracellular delivery of message RNA (mRNA) technique has ushered in a hopeful era with the successive authorization of two mRNA vaccines for the Coronavirus disease-19 (COVID-19) pandemic.
Yin Li +6 more
doaj +3 more sources
Lipid nanoparticle-targeted mRNA therapy as a treatment for the inherited metabolic liver disorder arginase deficiency. [PDF]
Proc Natl Acad Sci U S A, 2019 Significance Systemically administered lipid nanoparticles (LNPs) targeting the liver were able to express the cytoplasmic enzyme arginase 1 (ARG1) in a conditional knockout model of ARG1 deficiency.
Truong B +8 more
europepmc +2 more sources
Arginase I mRNA therapy - a novel approach to rescue arginase 1 enzyme deficiency. [PDF]
RNA Biol, 2018 Arginase I (ARG1) deficiency is an autosomal recessive urea cycle disorder, caused by deficiency of the enzyme Arginase I, resulting in accumulation of arginine in blood.
Asrani KH +3 more
europepmc +2 more sources
Nanoformulations targeting immune cells for cancer therapy: mRNA therapeutics
Bioactive Materials, 2023 The approved worldwide use of two messenger RNA (mRNA) vaccines (BNT162b2 and mRNA-1273) in late 2020 has proven the remarkable success of mRNA therapeutics together with lipid nanoformulation technology in protecting people against coronaviruses during ...
Wei Yang +6 more
doaj +2 more sources
Boosting ribosomal translation via ionizable lipid-hydrogel microplexes for localized mRNA therapy [PDF]
Bioactive MaterialsmRNA therapy holds immense promise for regenerative medicine; however, localized endoplasmic reticulum stress (ERS) in damaged tissues can impair the critical process of ribosomal translation.
Yanyang Chen +7 more
doaj +2 more sources
G6PC mRNA Therapy Positively Regulates Fasting Blood Glucose and Decreases Liver Abnormalities in a Mouse Model of Glycogen Storage Disease 1a. [PDF]
Mol Ther, 2018 Glycogen storage disease type Ia (GSD1a) is an inherited metabolic disorder caused by the deficiency of glucose-6-phosphatase (G6Pase). GSD1a is associated with life-threatening hypoglycemia and long-term liver and renal complications.
Roseman DS +7 more
europepmc +2 more sources