4.7 Article

ASL mRNA-LNP Therapeutic for the Treatment of Argininosuccinic Aciduria Enables Survival Benefit in a Mouse Model

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。
Review Biotechnology & Applied Microbiology

COVID-19 mRNA vaccines: Platforms and current developments

Gabor Tamas Szabo et al.

Summary: Since the discovery of the advantages of mRNA vaccines nearly 30 years ago, significant progress has been made in this field. The development of COVID-19 vaccines using mRNA technology has achieved unprecedented success globally, demonstrating the safety and effectiveness of mRNA vaccines in combating COVID-19 and highlighting their potential for other infectious diseases and medical conditions.

MOLECULAR THERAPY (2022)

Article Gastroenterology & Hepatology

Corticosteroid suppresses urea-cycle-related gene expressions in ornithine transcarbamylase deficiency

Koji Imoto et al.

Summary: This study investigated the mechanism of hyperammonemia induced by corticosteroid administration in a mouse model of OTCD. The results showed that corticosteroids induced hyperammonemia in OTCD mice by both muscle catabolism and suppression of gene expressions related to the urea cycle. Early intervention with renal replacement therapy should be considered in corticosteroid-induced UCD patients to prevent brain injuries or fatal outcomes.

BMC GASTROENTEROLOGY (2022)

Review Biotechnology & Applied Microbiology

Quality by Design for enabling RNA platform production processes

Simon Daniel et al.

Summary: RNA-based products play a crucial role in global vaccination, infectious disease control, and future therapy development. The assessment of critical quality attributes and product-process interactions can contribute to a robust Quality by Design framework for the development and control of manufacturing processes.

TRENDS IN BIOTECHNOLOGY (2022)

Review Immunology

Innate immune mechanisms of mRNA vaccines

Rein Verbeke et al.

Summary: The article reviews the use of lipid nanoparticle-encapsulated mRNA platform in COVID-19 vaccines and discusses the mechanism of innate immune activation induced by mRNA vaccines. The study highlights the role of LNP carrier as a potent adjuvant for this novel vaccine platform.

IMMUNITY (2022)

Review Immunology

Innate sensing of mRNA vaccines

Christine Wuebben et al.

Summary: The success of RNA applications, such as mRNA vaccines and RNA therapeutics, holds great promise for future drug development. Understanding innate RNA sensing can lead to further improvements in mRNA vaccines and RNA therapeutics.

CURRENT OPINION IN IMMUNOLOGY (2022)

Review Biochemistry & Molecular Biology

mRNA therapeutics in cancer immunotherapy

Jan D. Beck et al.

Summary: Synthetic mRNA serves as a versatile template for protein synthesis and has a wide range of pharmaceutical applications, including cancer immunotherapy. Strategies such as stimulating pattern recognition receptors and nucleoside modification enhance the effectiveness and safety of mRNA vaccines.

MOLECULAR CANCER (2021)

Review Biochemistry & Molecular Biology

Non-Immunotherapy Application of LNP-mRNA: Maximizing Efficacy and Safety

Irena Vlatkovic

Summary: LNP-mRNA vaccines, with their advantages of fast engineering and efficient manufacturing, have shown potential in combating SARS-CoV-2 virus. However, in protein replacement therapy, it is crucial to avoid immune activation.

BIOMEDICINES (2021)

Article Gastroenterology & Hepatology

Liver Transplantation in Children with Urea Cycle Disorders: The Importance of Minimizing Waiting Time

Ioannis A. Ziogas et al.

Summary: Liver transplantation is an effective treatment for children with urea cycle disorders, correcting enzymatic defects and preventing neurologic injury. Factors such as increasing weight, male sex, and specific UCD diagnoses are associated with reduced risk of graft loss, while longer waitlist time and being male increase the odds of long-term cognitive delay. Early evaluation for LT in children with UCDs is recommended to optimize cognitive outcomes and prevent progressive neurologic injury.

LIVER TRANSPLANTATION (2021)

Review Pharmacology & Pharmacy

Lipid nanoparticles for nucleic acid delivery: Current perspectives

Eleni Samaridou et al.

ADVANCED DRUG DELIVERY REVIEWS (2020)

Article Multidisciplinary Sciences

COVID-19 vaccine BNT162b1 elicits human antibody and TH1 T cell responses

Ugur Sahin et al.

NATURE (2020)

Article Genetics & Heredity

Late-onset argininosuccinic aciduria associated with hyperammonemia triggered by influenza infection in an adolescent: A case report

Yoshimitsu Osawa et al.

MOLECULAR GENETICS AND METABOLISM REPORTS (2020)

Article Endocrinology & Metabolism

Suggested guidelines for the diagnosis and management of urea cycle disorders: First revision

Johannes Haberle et al.

JOURNAL OF INHERITED METABOLIC DISEASE (2019)

Article Medicine, Research & Experimental

A Facile Method for the Removal of dsRNA Contaminant from In Vitro-Transcribed mRNA

Markus Baiersdoerfer et al.

MOLECULAR THERAPY-NUCLEIC ACIDS (2019)

Article Biotechnology & Applied Microbiology

Improving mRNA-Based Therapeutic Gene Delivery by Expression-Augmenting 3′ UTRs Identified by Cellular Library Screening

Alexandra G. Orlandini von Niessen et al.

MOLECULAR THERAPY (2019)

Article Biotechnology & Applied Microbiology

Targeted mRNA Therapy for Ornithine Transcarbamylase Deficiency

Mary G. Prieve et al.

MOLECULAR THERAPY (2018)

Review Medicine, Research & Experimental

Emerging Issues in AAV-Mediated In Vivo Gene Therapy

Pasqualina Colella et al.

MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2018)

Article Endocrinology & Metabolism

Adeno-associated viral gene therapy corrects a mouse model of argininosuccinic aciduria

Scott N. Ashley et al.

MOLECULAR GENETICS AND METABOLISM (2018)

Article Multidisciplinary Sciences

Argininosuccinic aciduria fosters neuronal nitrosative stress reversed by Asl gene transfer

Julien Baruteau et al.

NATURE COMMUNICATIONS (2018)

Review Biotechnology & Applied Microbiology

Recombinant Adeno-Associated Viral Integration and Genotoxicity: Insights from Animal Models

Randy J. Chandler et al.

HUMAN GENE THERAPY (2017)

Editorial Material Biotechnology & Applied Microbiology

Genotoxicity in Mice Following AAV Gene Delivery: A Safety Concern for Human Gene Therapy?

Randy J. Chandler et al.

MOLECULAR THERAPY (2016)

Review Multidisciplinary Sciences

Translational control by 5′-untranslated regions of eukaryotic mRNAs

Alan G. Hinnebusch et al.

SCIENCE (2016)

Article Medicine, Research & Experimental

Vector design influences hepatic genotoxicity after adeno-associated virus gene therapy

Randy J. Chandler et al.

JOURNAL OF CLINICAL INVESTIGATION (2015)

Review Genetics & Heredity

Argininosuccinic aciduria: from a monogenic to a complex disorder

Ayelet Erez

GENETICS IN MEDICINE (2013)

Article Endocrinology & Metabolism

The incidence of urea cycle disorders

Marshall L. Summar et al.

MOLECULAR GENETICS AND METABOLISM (2013)

Article Biochemistry & Molecular Biology

Ammonia triggers neuronal disinhibition and seizures by impairing astrocyte potassium buffering

Vinita Rangroo Thrane et al.

NATURE MEDICINE (2013)

Review Genetics & Heredity

Suggested guidelines for the diagnosis and management of urea cycle disorders

Johannes Haeberle et al.

ORPHANET JOURNAL OF RARE DISEASES (2012)

Article Biochemistry & Molecular Biology

Requirement of argininosuccinate lyase for systemic nitric oxide production

Ayelet Erez et al.

NATURE MEDICINE (2011)

Article Gastroenterology & Hepatology

Liver transplantation for argininosuccinic aciduria: Clinical, biochemical, and metabolic outcome

Tanya Newnham et al.

LIVER TRANSPLANTATION (2008)

Article Multidisciplinary Sciences

AAV vector integration sites in mouse hepatocellular carcinoma

Anthony Donsante et al.

SCIENCE (2007)

Article Chemistry, Multidisciplinary

Synthesis and characterization of novel poly(ethylene glycol)-lipid conjugates suitable for use in drug delivery

James Heyes et al.

JOURNAL OF CONTROLLED RELEASE (2006)

Article Nutrition & Dietetics

Arginine metabolic enzymes, nitric oxide and infection

M Mori et al.

JOURNAL OF NUTRITION (2004)

Article Multidisciplinary Sciences

Distribution of nitric oxide synthase in normal and cirrhotic human liver

L McNaughton et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2002)

Review Biochemistry & Molecular Biology

Urea-cycle disorders as a paradigm for inborn errors of hepatocyte metabolism

A Mian et al.

TRENDS IN MOLECULAR MEDICINE (2002)