标题
mRNA in the Context of Protein Replacement Therapy
作者
关键词
-
出版物
Pharmaceutics
Volume 15, Issue 1, Pages 166
出版商
MDPI AG
发表日期
2023-01-04
DOI
10.3390/pharmaceutics15010166
参考文献
相关参考文献
注意：仅列出部分参考文献，下载原文获取全部文献信息。- Next generation self-replicating RNA vectors for vaccines and immunotherapies
- (2022) Parinaz Aliahmad et al. CANCER GENE THERAPY
- What Is Cystic Fibrosis?
- (2022) Tori M. Endres et al. JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION
- Lyophilization provides long-term stability for a lipid nanoparticle-formulated, nucleoside-modified mRNA vaccine
- (2022) Hiromi Muramatsu et al. MOLECULAR THERAPY
- Ribozyme Assays to Quantify the Capping Efficiency of In Vitro-Transcribed mRNA
- (2022) Irena Vlatkovic et al. Pharmaceutics
- Quality by Design for enabling RNA platform production processes
- (2022) Simon Daniel et al. TRENDS IN BIOTECHNOLOGY
- Development of an mRNA replacement therapy for phenylketonuria
- (2022) Carlos G. Perez-Garcia et al. Molecular Therapy-Nucleic Acids
- Making COVID-19 mRNA vaccines accessible: challenges resolved
- (2022) Sarfaraz K. Niazi Expert Review of Vaccines
- mRNA-based modalities for infectious disease management
- (2022) Mengjie Zhang et al. Nano Research
- Spotlight on Givosiran as a Treatment Option for Adults with Acute Hepatic Porphyria: Design, Development, and Place in Therapy
- (2022) Chaudry Nasir Majeed et al. Drug Design Development and Therapy
- Gram‐Scale mRNA Production Using a 250‐mL Single‐Use Bioreactor
- (2022) Janja Skok et al. CHEMIE INGENIEUR TECHNIK
- Self-Amplifying RNA Approach for Protein Replacement Therapy
- (2022) Dimitri Papukashvili et al. INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
- Payload distribution and capacity of mRNA lipid nanoparticles
- (2022) Sixuan Li et al. Nature Communications
- The landscape of mRNA nanomedicine
- (2022) Xiangang Huang et al. NATURE MEDICINE
- Novel lipid combination for delivery of plasmid DNA to immune cells in the spleen
- (2021) Seigo Kimura et al. JOURNAL OF CONTROLLED RELEASE
- BNT162b vaccines protect rhesus macaques from SARS-CoV-2
- (2021) Annette B. Vogel et al. NATURE
- mRNA vaccines manufacturing: Challenges and bottlenecks
- (2021) Sara Sousa Rosa et al. VACCINE
- The Limitless Future of RNA Therapeutics
- (2021) Tulsi Ram Damase et al. Frontiers in Bioengineering and Biotechnology
- Strategies for controlling the innate immune activity of conventional and self-amplifying mRNA therapeutics: Getting the message across
- (2021) An-Katrien Minnaert et al. ADVANCED DRUG DELIVERY REVIEWS
- An overview of rational design of mRNA-based therapeutics and vaccines
- (2021) Kenneth K.W. To et al. Expert Opinion on Drug Discovery
- Modifications of mRNA vaccine structural elements for improving mRNA stability and translation efficiency
- (2021) Sun Chang Kim et al. Molecular & Cellular Toxicology
- BNT162b2 vaccine induces neutralizing antibodies and poly-specific T cells in humans
- (2021) Ugur Sahin et al. NATURE
- Low-dose mRNA-1273 COVID-19 vaccine generates durable memory enhanced by cross-reactive T cells
- (2021) Jose Mateus et al. SCIENCE
- Lipid nanoparticles for mRNA delivery
- (2021) Xucheng Hou et al. Nature Reviews Materials
- Pharma 4.0 Continuous mRNA Drug Products Manufacturing
- (2021) Andreas Ouranidis et al. Pharmaceutics
- Challenges of Storage and Stability of mRNA-Based COVID-19 Vaccines
- (2021) Mohammad N. Uddin et al. Vaccines
- Ionization and structural properties of mRNA lipid nanoparticles influence expression in intramuscular and intravascular administration
- (2021) Manuel J. Carrasco et al. Communications Biology
- Roles of mRNA poly(A) tails in regulation of eukaryotic gene expression
- (2021) Lori A. Passmore et al. NATURE REVIEWS MOLECULAR CELL BIOLOGY
- mRNA, a Revolution in Biomedicine
- (2021) Bruno Baptista et al. Pharmaceutics
- mRNA Therapeutic Modalities Design, Formulation and Manufacturing under Pharma 4.0 Principles
- (2021) Andreas Ouranidis et al. Biomedicines
- Nanomedicines to Deliver mRNA: State of the Art and Future Perspectives
- (2020) Itziar Gómez-Aguado et al. Nanomaterials
- Deconvoluting Lipid Nanoparticle Structure for Messenger RNA Delivery
- (2020) Yulia Eygeris et al. NANO LETTERS
- Characterization of spike glycoprotein of SARS-CoV-2 on virus entry and its immune cross-reactivity with SARS-CoV
- (2020) Xiuyuan Ou et al. Nature Communications
- SARS-CoV-2 mRNA vaccine design enabled by prototype pathogen preparedness
- (2020) Kizzmekia S. Corbett et al. NATURE
- Long-term storage of lipid-like nanoparticles for mRNA delivery
- (2020) Pengxuan Zhao et al. Bioactive Materials
- Treating Cystic Fibrosis with mRNA and CRISPR
- (2020) Alejandro Da Silva Sanchez et al. HUMAN GENE THERAPY
- Synthetic mRNA Encoding VEGF-A in Patients Undergoing Coronary Artery Bypass Grafting: Design of a Phase 2a Clinical Trial
- (2020) Vesa Anttila et al. Molecular Therapy-Methods & Clinical Development
- Addressing the Cold Reality of mRNA Vaccine Stability
- (2020) Daan J.A. Crommelin et al. JOURNAL OF PHARMACEUTICAL SCIENCES
- Efficacy and Safety of the mRNA-1273 SARS-CoV-2 Vaccine
- (2020) Lindsey R. Baden et al. NEW ENGLAND JOURNAL OF MEDICINE
- RNA Modifications Modulate Activation of Innate Toll-Like Receptors
- (2019) Isabel Freund et al. Genes
- Improved Efficacy in a Fabry Disease Model Using a Systemic mRNA Liver Depot System as Compared to Enzyme Replacement Therapy
- (2019) Frank DeRosa et al. MOLECULAR THERAPY
- Delivering the Messenger: Advances in Technologies for Therapeutic mRNA Delivery
- (2019) Piotr S. Kowalski et al. MOLECULAR THERAPY
- Messenger RNA therapy for rare genetic metabolic diseases
- (2019) Pedro Berraondo et al. GUT
- Phenylketonuria: Current Treatments and Future Developments
- (2019) Uta Lichter-Konecki et al. DRUGS
- Nanoparticles Containing Oxidized Cholesterol Deliver mRNA to the Liver Microenvironment at Clinically Relevant Doses
- (2019) Kalina Paunovska et al. ADVANCED MATERIALS
- Inotersen: new promise for the treatment of hereditary transthyretin amyloidosis
- (2019) Veena Mathew et al. Drug Design Development and Therapy
- Volanesorsen: First Global Approval
- (2019) Julia Paik et al. DRUGS
- Correction of bleeding in experimental severe hemophilia A by systemic delivery of factor VIII-encoding mRNA
- (2019) Jules Russick et al. HAEMATOLOGICA
- Messenger RNA therapy as an option for treating metabolic disorders
- (2019) Randy J. Chandler PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Lipid nanoparticle-targeted mRNA therapy as a treatment for the inherited metabolic liver disorder arginase deficiency
- (2019) Brian Truong et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- mRNA structure regulates protein expression through changes in functional half-life
- (2019) David M. Mauger et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Tailoring mRNA Vaccine to Balance Innate/Adaptive Immune Response
- (2019) Sergio Linares-Fernández et al. TRENDS IN MOLECULAR MEDICINE
- GC content shapes mRNA storage and decay in human cells
- (2019) Maïté Courel et al. eLife
- Analyzing 2000 in Vivo Drug Delivery Data Points Reveals Cholesterol Structure Impacts Nanoparticle Delivery
- (2018) Kalina Paunovska et al. ACS Nano
- Poly A tail length analysis of in vitro transcribed mRNA by LC-MS
- (2018) Michael Beverly et al. ANALYTICAL AND BIOANALYTICAL CHEMISTRY
- Lipid Polymer Hybrid Nanomaterials for mRNA Delivery
- (2018) Weiyu Zhao et al. Cellular and Molecular Bioengineering
- Lipid Nanoparticle-Delivered Chemically Modified mRNA Restores Chloride Secretion in Cystic Fibrosis
- (2018) Ema Robinson et al. MOLECULAR THERAPY
- Lipid Nanoparticles Enabling Gene Therapies: From Concepts to Clinical Utility
- (2018) Jayesh A. Kulkarni et al. Nucleic Acid Therapeutics
- Biocompatible, Purified VEGF-A mRNA Improves Cardiac Function after Intracardiac Injection 1 Week Post-myocardial Infarction in Swine
- (2018) Leif Carlsson et al. Molecular Therapy-Methods & Clinical Development
- What Are 3′ UTRs Doing?
- (2018) Christine Mayr Cold Spring Harbor Perspectives in Biology
- Delivery of mRNA Therapeutics for the Treatment of Hepatic Diseases
- (2018) Zeljka Trepotec et al. MOLECULAR THERAPY
- Improving mRNA-Based Therapeutic Gene Delivery by Expression-Augmenting 3′ UTRs Identified by Cellular Library Screening
- (2018) Alexandra G. Orlandini von Niessen et al. MOLECULAR THERAPY
- mRNA-Based Protein Replacement Therapy for the Heart
- (2018) Ajit Magadum et al. MOLECULAR THERAPY
- mRNA decapping: finding the right structures
- (2018) Clément Charenton et al. PHILOSOPHICAL TRANSACTIONS OF THE ROYAL SOCIETY B-BIOLOGICAL SCIENCES
- Chemically modified hCFTR mRNAs recuperate lung function in a mouse model of cystic fibrosis
- (2018) A. K. M. Ashiqul Haque et al. Scientific Reports
- Modified VEGF-A mRNA induces sustained multifaceted microvascular response and accelerates diabetic wound healing
- (2018) Naidi Sun et al. Scientific Reports
- Safety and immunogenicity of a mRNA rabies vaccine in healthy adults: an open-label, non-randomised, prospective, first-in-human phase 1 clinical trial
- (2017) Martin Alberer et al. LANCET
- Optimizing Cardiac Delivery of Modified mRNA
- (2017) Nishat Sultana et al. MOLECULAR THERAPY
- Systemic delivery of factor IX messenger RNA for protein replacement therapy
- (2017) Suvasini Ramaswamy et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Eteplirsen in the treatment of Duchenne muscular dystrophy
- (2017) Kenji Rowel Lim et al. Drug Design Development and Therapy
- Surface De-PEGylation Controls Nanoparticle-Mediated siRNA Delivery In Vitro and In Vivo
- (2017) Xi Zhu et al. Theranostics
- pEVL: A Linear Plasmid for Generating mRNA IVT Templates With Extended Encoded Poly(A) Sequences
- (2016) Alexandra E Grier et al. Molecular Therapy-Nucleic Acids
- N 1 -methylpseudouridine-incorporated mRNA outperforms pseudouridine-incorporated mRNA by providing enhanced protein expression and reduced immunogenicity in mammalian cell lines and mice
- (2015) Oliwia Andries et al. JOURNAL OF CONTROLLED RELEASE
- Genotype–Phenotype Correlations in Ornithine Transcarbamylase Deficiency: A Mutation Update
- (2015) Ljubica Caldovic et al. Journal of Genetics and Genomics
- Arginase-1 deficiency
- (2015) Yuan Yan Sin et al. JOURNAL OF MOLECULAR MEDICINE-JMM
- mRNA-based therapeutics — developing a new class of drugs
- (2014) Ugur Sahin et al. NATURE REVIEWS DRUG DISCOVERY
- Determinants and implications of mRNA poly(A) tail size – Does this protein make my tail look big?
- (2014) Aimee L. Jalkanen et al. SEMINARS IN CELL & DEVELOPMENTAL BIOLOGY
- Large-scale purification of pharmaceutical-grade plasmid DNA using tangential flow filtration and multi-step chromatography
- (2013) Bo Sun et al. JOURNAL OF BIOSCIENCE AND BIOENGINEERING
- Modified mRNA directs the fate of heart progenitor cells and induces vascular regeneration after myocardial infarction
- (2013) Lior Zangi et al. NATURE BIOTECHNOLOGY
- Efficient genome editing in zebrafish using a CRISPR-Cas system
- (2013) Woong Y Hwang et al. NATURE BIOTECHNOLOGY
- Protein Replacement Therapies for Rare Diseases: A Breeze for Regulatory Approval?
- (2013) J. A. Gorzelany et al. Science Translational Medicine
- Increased Erythropoiesis in Mice Injected With Submicrogram Quantities of Pseudouridine-containing mRNA Encoding Erythropoietin
- (2012) Katalin Karikó et al. MOLECULAR THERAPY
- Protective efficacy of in vitro synthesized, specific mRNA vaccines against influenza A virus infection
- (2012) Benjamin Petsch et al. NATURE BIOTECHNOLOGY
- Nonviral delivery of self-amplifying RNA vaccines
- (2012) A. J. Geall et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Developing mRNA-vaccine technologies
- (2012) Thomas Schlake et al. RNA Biology
- Phenylalanine hydroxylase deficiency
- (2011) John J. Mitchell et al. GENETICS IN MEDICINE
- Genome Analysis Reveals Interplay between 5′UTR Introns and Nuclear mRNA Export for Secretory and Mitochondrial Genes
- (2011) Can Cenik et al. PLoS Genetics
- RNA Isolation from Mammalian Cells Using Porous Polymer Monoliths: An Approach for High-Throughput Automation
- (2010) Anirban Chatterjee et al. ANALYTICAL CHEMISTRY
- Intranodal Vaccination with Naked Antigen-Encoding RNA Elicits Potent Prophylactic and Therapeutic Antitumoral Immunity
- (2010) S. Kreiter et al. CANCER RESEARCH
- A Role for Codon Order in Translation Dynamics
- (2010) Gina Cannarozzi et al. CELL
- Direct Injection of Protamine-protected mRNA: Results of a Phase 1/2 Vaccination Trial in Metastatic Melanoma Patients
- (2009) Benjamin Weide et al. JOURNAL OF IMMUNOTHERAPY
- Optimizing the use of sapropterin (BH4) in the management of phenylketonuria
- (2009) Nenad Blau et al. MOLECULAR GENETICS AND METABOLISM
- Defining tetrahydrobiopterin (BH4)-responsiveness in PKU
- (2008) Nenad Blau JOURNAL OF INHERITED METABOLIC DISEASE
- Incorporation of Pseudouridine Into mRNA Yields Superior Nonimmunogenic Vector With Increased Translational Capacity and Biological Stability
- (2008) Katalin Karikó et al. MOLECULAR THERAPY
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