Systemic modified messenger RNA for replacement therapy in alpha 1-antitrypsin deficiency

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

A Novel Amino Lipid Series for mRNA Delivery: Improved Endosomal Escape and Sustained Pharmacology and Safety in Non-human Primates

(2018) Staci Sabnis et al.   MOLECULAR THERAPY

Systemic messenger RNA as an etiological treatment for acute intermittent porphyria

(2018) Lei Jiang et al.   NATURE MEDICINE

Bone marrow stem cell therapy partially ameliorates pathological consequences in livers of mice expressing mutant human α1-antitrypsin

(2017) Prakash Baligar et al.   HEPATOLOGY

Exogenous alpha 1-antitrypsin down-regulates SERPINA1 expression

(2017) Ahmad Karadagi et al.   PLoS One

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

Systemic Messenger RNA Therapy as a Treatment for Methylmalonic Acidemia

(2017) Ding An et al.   Cell Reports

Fluorescent proteins such as eGFP lead to catalytic oxidative stress in cells

(2017) Douglas Ganini et al.   Redox Biology

Therapeutic efficacy in a hemophilia B model using a biosynthetic mRNA liver depot system

(2016) F DeRosa et al.   GENE THERAPY

Cellular GFP Toxicity and Immunogenicity: Potential Confounders in in Vivo Cell Tracking Experiments

(2016) Amir Mehdi Ansari et al.   Stem Cell Reviews and Reports

Cellular GFP Toxicity and Immunogenicity: Potential Confounders in in Vivo Cell Tracking Experiments

(2016) Amir Mehdi Ansari et al.   Stem Cell Reviews and Reports

α1-Antitrypsin deficiency

(2016) Catherine M. Greene et al.   Nature Reviews Disease Primers

In VitroEvaluation of a Novel mRNA-Based Therapeutic Strategy for the Treatment of Patients Suffering from Alpha-1-Antitrypsin Deficiency

(2015) Tatjana Michel et al.   Nucleic Acid Therapeutics

Immune-modulating effects of alpha-1 antitrypsin

(2014) Mario R. Ehlers   BIOLOGICAL CHEMISTRY

Current status of gene therapy for α-1 antitrypsin deficiency

(2014) Heather S Loring et al.   EXPERT OPINION ON BIOLOGICAL THERAPY

Non-viral vectors for gene-based therapy

(2014) Hao Yin et al.   NATURE REVIEWS GENETICS

Alpha-1 antitrypsin and liver disease: mechanisms of injury and novel interventions

(2014) Jeffrey H Teckman et al.   Expert Review of Gastroenterology & Hepatology

Shielding of Lipid Nanoparticles for siRNA Delivery: Impact on Physicochemical Properties, Cytokine Induction, and Efficacy

(2014) Varun Kumar et al.   Molecular Therapy-Nucleic Acids

The Electrophoretic α1-Globulin Pattern of Serum in α1-Antitrypsin Deficiency

(2013) Carl-Bertil Laurell et al.   COPD-Journal of Chronic Obstructive Pulmonary Disease

Hepatocyte transplantation for inherited metabolic diseases of the liver

(2013) C. Jorns et al.   JOURNAL OF INTERNAL MEDICINE

Outcomes for recipients of liver transplantation for alpha-1-antitrypsin deficiency-related cirrhosis

(2013) Elizabeth J. Carey et al.   LIVER TRANSPLANTATION

Biodegradable Lipids Enabling Rapidly Eliminated Lipid Nanoparticles for Systemic Delivery of RNAi Therapeutics

(2013) Martin A Maier et al.   MOLECULAR THERAPY

Augmentation therapy for alpha-1 antitrypsin deficiency: towards a personalised approach

(2013) Robert A Stockley et al.   Orphanet Journal of Rare Diseases

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(2012) Eli Lewis   MOLECULAR MEDICINE

NIH Image to ImageJ: 25 years of image analysis

(2012) Caroline A Schneider et al.   NATURE METHODS

Alpha 1-antitrypsin reduces inflammation and enhances mouse pancreatic islet transplant survival

(2012) M. Koulmanda et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

A Review of α1-Antitrypsin Deficiency

(2011) James K. Stoller et al.   AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE

Development and Application of Purified Tissue Dissociation Enzyme Mixtures for Human Hepatocyte Isolation

(2011) Roberto Gramignoli et al.   CELL TRANSPLANTATION

The role of augmentation therapy in alpha-1 antitrypsin deficiency

(2011) F. Kueppers   CURRENT MEDICAL RESEARCH AND OPINION

Augmentation Therapy for α1Antitrypsin Deficiency: A Meta-Analysis

(2009) Kenneth R. Chapman et al.   COPD-Journal of Chronic Obstructive Pulmonary Disease

Incorporation of Pseudouridine Into mRNA Yields Superior Nonimmunogenic Vector With Increased Translational Capacity and Biological Stability

(2008) Katalin Karikó et al.   MOLECULAR THERAPY