Pulmonary transplantation of alpha-1 antitrypsin (AAT)-transgenic macrophages provides a source of functional human AAT in vivo

Approach for Profiling of Glycosphingolipid Glycosylation by Multiplexed Capillary Gel Electrophoresis Coupled to Laser-Induced Fluorescence Detection To Identify Cell-Surface Markers of Human Pluripotent Stem Cells and Derived Cardiomyocytes

(2019) Charlotte Rossdam et al.   ANALYTICAL CHEMISTRY

Pulmonary Transplantation of Human iPSC-derived Macrophages Ameliorates Pulmonary Alveolar Proteinosis

(2018) Christine Happle et al.   AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE

The murine lung as a factory to produce secreted intrapulmonary and circulatory proteins

(2018) Michael C. Paul-Smith et al.   GENE THERAPY

Editing out fiveSerpina1paralogs to create a mouse model of genetic emphysema

(2018) Florie Borel et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Alpha 1 antitrypsin to treat lung disease in alpha 1 antitrypsin deficiency: recent developments and clinical implications

(2018) Kenneth R Chapman et al.   International Journal of Chronic Obstructive Pulmonary Disease

iPSC-Derived Macrophages Effectively Treat Pulmonary Alveolar Proteinosis in Csf2rb-Deficient Mice

(2018) Adele Mucci et al.   Stem Cell Reports

A murine model of elastase- and cigarette smoke-induced emphysema

(2017) Rubia Rodrigues et al.   Jornal Brasileiro de Pneumologia

5 Year Expression and Neutrophil Defect Repair after Gene Therapy in Alpha-1 Antitrypsin Deficiency

(2017) Christian Mueller et al.   MOLECULAR THERAPY

Function and Safety of Lentivirus-Mediated Gene Transfer for CSF2RA-Deficiency

(2017) Miriam Hetzel et al.   Human Gene Therapy Methods

TALEN-mediated functional correction of human iPSC-derived macrophages in context of hereditary pulmonary alveolar proteinosis

(2017) Alexandra Kuhn et al.   Scientific Reports

Yolk Sac Macrophages, Fetal Liver, and Adult Monocytes Can Colonize an Empty Niche and Develop into Functional Tissue-Resident Macrophages

(2016) Lianne van de Laar et al.   IMMUNITY

Lentiviral hematopoietic stem cell gene therapy for X-linked severe combined immunodeficiency

(2016) Suk See De Ravin et al.   Science Translational Medicine

Intravenous augmentation treatment and lung density in severe α1 antitrypsin deficiency (RAPID): a randomised, double-blind, placebo-controlled trial

(2015) Kenneth R Chapman et al.   LANCET

A minimal ubiquitous chromatin opening element (UCOE) effectively prevents silencing of juxtaposed heterologous promoters by epigenetic remodeling in multipotent and pluripotent stem cells

(2015) Uta Müller-Kuller et al.   NUCLEIC ACIDS RESEARCH

Unopposed Cathepsin G, Neutrophil Elastase, and Proteinase 3 Cause Severe Lung Damage and Emphysema

(2014) Nicolas Guyot et al.   AMERICAN JOURNAL OF PATHOLOGY

Pulmonary macrophage transplantation therapy

(2014) Takuji Suzuki et al.   NATURE

A Modified γ-Retrovirus Vector for X-Linked Severe Combined Immunodeficiency

(2014) Salima Hacein-Bey-Abina et al.   NEW ENGLAND JOURNAL OF MEDICINE

Pulmonary transplantation of macrophage progenitors as effective and long-lasting therapy for hereditary pulmonary alveolar proteinosis

(2014) C. Happle et al.   Science Translational Medicine

Phase I/II Study of Intrapleural Administration of a Serotype rh.10 Replication-Deficient Adeno-Associated Virus Gene Transfer Vector Expressing the Human α1-Antitrypsin cDNA to Individuals with α1-Antitrypsin Deficiency

(2014) Maria J. Chiuchiolo et al.   Human Gene Therapy Clinical Development

Flow Cytometric Analysis of Macrophages and Dendritic Cell Subsets in the Mouse Lung

(2013) Alexander V. Misharin et al.   AMERICAN JOURNAL OF RESPIRATORY CELL AND MOLECULAR BIOLOGY

Biosafety Features of Lentiviral Vectors

(2013) Axel Schambach et al.   HUMAN GENE THERAPY

Alveolar macrophages develop from fetal monocytes that differentiate into long-lived cells in the first week of life via GM-CSF

(2013) Martin Guilliams et al.   JOURNAL OF EXPERIMENTAL MEDICINE

Anti-inflammatory and immunomodulatory properties of  1-antitrypsin without inhibition of elastase

(2013) D. Jonigk et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Intrapleural Administration of an AAVrh.10 Vector Coding for Human α1-Antitrypsin for the Treatment of α1-Antitrypsin Deficiency

(2013) Maria J. Chiuchiolo et al.   Human Gene Therapy Clinical Development

Fate Mapping Reveals Origins and Dynamics of Monocytes and Tissue Macrophages under Homeostasis

(2012) Simon Yona et al.   IMMUNITY

Phase 2 Clinical Trial of a Recombinant Adeno-Associated Viral Vector Expressing α1-Antitrypsin: Interim Results

(2011) Terence R. Flotte et al.   HUMAN GENE THERAPY

The discovery of α1-antitrypsin and its role in health and disease

(2011) Sabina M. Janciauskiene et al.   RESPIRATORY MEDICINE

Fate Mapping Analysis Reveals That Adult Microglia Derive from Primitive Macrophages

(2010) F. Ginhoux et al.   SCIENCE

Amelioration of emphysema in mice through lentiviral transduction of long-lived pulmonary alveolar macrophages

(2009) Andrew A. Wilson et al.   JOURNAL OF CLINICAL INVESTIGATION

Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy

(2009) M. L. Brantly et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Sustained Expression of α1-Antitrypsin after Transplantation of Manipulated Hematopoietic Stem Cells

(2008) Andrew A. Wilson et al.   AMERICAN JOURNAL OF RESPIRATORY CELL AND MOLECULAR BIOLOGY

Treatment of Leber Congenital Amaurosis Due to RPE65 Mutations by Ocular Subretinal Injection of Adeno-Associated Virus Gene Vector: Short-Term Results of a Phase I Trial

(2008) William W. Hauswirth et al.   HUMAN GENE THERAPY