Current Status and Future Directions of Gene and Cell Therapy for Cystic Fibrosis

Effects of fibroblast growth factors on the differentiation of the pulmonary progenitors from murine embryonic stem cells

(2010) Yuan Min Lin et al.   EXPERIMENTAL LUNG RESEARCH

Emerging therapies for cystic fibrosis lung disease

(2010) Hartmut Grasemann et al.   EXPERT OPINION ON EMERGING DRUGS

Transgene Loss and Changes in the Promoter Methylation Status as Determinants for Expression Duration in Nonviral Gene Transfer for Factor IX

(2010) Jörg Schüttrumpf et al.   HUMAN GENE THERAPY

Respiratory Syncytial Virus Engineered To Express the Cystic Fibrosis Transmembrane Conductance Regulator Corrects the Bioelectric Phenotype of Human Cystic Fibrosis Airway Epithelium In Vitro

(2010) A. R. Kwilas et al.   JOURNAL OF VIROLOGY

Transplantation of Human Embryonic Stem Cell–Derived Alveolar Epithelial Type II Cells Abrogates Acute Lung Injury in Mice

(2010) Dachun Wang et al.   MOLECULAR THERAPY

Embryonic Stem Cells and Repair of Lung Injury

(2010) Daniel J Weiss et al.   MOLECULAR THERAPY

Optimization of Targeted Cell Replacement Therapy: A New Approach for Lung Disease

(2010) Pascal Duchesneau et al.   MOLECULAR THERAPY

Toward Gene Therapy for Cystic Fibrosis Using a Lentivirus Pseudotyped With Sendai Virus Envelopes

(2010) Katsuyuki Mitomo et al.   MOLECULAR THERAPY

Expression of Human α1-Antitrypsin in Mice and Dogs Following AAV6 Vector-mediated Gene Transfer to the Lungs

(2010) Christine L Halbert et al.   MOLECULAR THERAPY

Tissue-Engineered Lungs for in Vivo Implantation

(2010) T. H. Petersen et al.   SCIENCE

Cystic Fibrosis Pigs Develop Lung Disease and Exhibit Defective Bacterial Eradication at Birth

(2010) D. A. Stoltz et al.   Science Translational Medicine

Generation of Transgene-Free Lung Disease-Specific Human Induced Pluripotent Stem Cells Using a Single Excisable Lentiviral Stem Cell Cassette

(2010) Aba Somers et al.   STEM CELLS

Limitations of the Murine Nose in the Development of Nonviral Airway Gene Transfer

(2009) Uta Griesenbach et al.   AMERICAN JOURNAL OF RESPIRATORY CELL AND MOLECULAR BIOLOGY

The role of doxorubicin in non-viral gene transfer in the lung

(2009) Uta Griesenbach et al.   BIOMATERIALS

The use of carboxymethylcellulose gel to increase non-viral gene transfer in mouse airways

(2009) Uta Griesenbach et al.   BIOMATERIALS

Non-viral vectors in cystic fibrosis gene therapy: recent developments and future prospects

(2009) IA Pringle et al.   EXPERT OPINION ON BIOLOGICAL THERAPY

Progress and Prospects: The design and production of plasmid vectors

(2009) D R Gill et al.   GENE THERAPY

Developing Cell Therapy Techniques for Respiratory Disease: Intratracheal Delivery of Genetically Engineered Stem Cells in a Murine Model of Airway Injury

(2009) Anne-Laure Leblond et al.   HUMAN GENE THERAPY

Identification and Functional Characterization of Cytoplasmic Determinants of Plasmid DNA Nuclear Import

(2009) Felix M. Munkonge et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

The genotoxic potential of retroviral vectors is strongly modulated by vector design and integration site selection in a mouse model of HSC gene therapy

(2009) Eugenio Montini et al.   JOURNAL OF CLINICAL INVESTIGATION

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

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

Single-dose lentiviral gene transfer for lifetime airway gene expression

(2009) Alice G. Stocker et al.   JOURNAL OF GENE MEDICINE

Engraftment of Bone Marrow–derived Stem Cells to the Lung in a Model of Acute Respiratory Infection by Pseudomonas aeruginosa

(2009) Joanna Rejman et al.   MOLECULAR THERAPY

CFTR Delivery to 25% of Surface Epithelial Cells Restores Normal Rates of Mucus Transport to Human Cystic Fibrosis Airway Epithelium

(2009) Liqun Zhang et al.   PLOS BIOLOGY

Gene transfer to the lung: Lessons learned from more than 2 decades of CF gene therapy

(2008) Uta Griesenbach et al.   ADVANCED DRUG DELIVERY REVIEWS

Extracellular barriers in respiratory gene therapy

(2008) Niek Sanders et al.   ADVANCED DRUG DELIVERY REVIEWS

Ciliated epithelial cell lifespan in the mouse trachea and lung

(2008) Emma L. Rawlins et al.   AMERICAN JOURNAL OF PHYSIOLOGY-LUNG CELLULAR AND MOLECULAR PHYSIOLOGY

Lentiviral transduction of the murine lung provides efficient pseudotype and developmental stage-dependent cell-specific transgene expression

(2008) S M K Buckley et al.   GENE THERAPY

Adeno-associated virus–targeted disruption of the CFTR gene in cloned ferrets

(2008) Xingshen Sun et al.   JOURNAL OF CLINICAL INVESTIGATION

Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients

(2008) Steven J. Howe et al.   JOURNAL OF CLINICAL INVESTIGATION

Short hairpin RNAs against eotaxin or interleukin-5 decrease airway eosinophilia and hyper-responsiveness in a murine model of asthma

(2008) Hsin-Ying Huang et al.   JOURNAL OF GENE MEDICINE

Lentivirus Vector Can Be Readministered to Nasal Epithelia without Blocking Immune Responses

(2008) P. L. Sinn et al.   JOURNAL OF VIROLOGY

Clinical transplantation of a tissue-engineered airway

(2008) Paolo Macchiarini et al.   LANCET

Intra-amniotic Delivery of CFTR-expressing Adenovirus Does Not Reverse Cystic Fibrosis Phenotype in Inbred CFTR-knockout Mice

(2008) Suzanne MK Buckley et al.   MOLECULAR THERAPY

Enhanced Lung Gene Expression After Aerosol Delivery of Concentrated pDNA/PEI Complexes

(2008) Lee A Davies et al.   MOLECULAR THERAPY

Adenovirus-mediated In Utero Expression of CFTR Does Not Improve Survival of CFTR Knockout Mice

(2008) Lee A Davies et al.   MOLECULAR THERAPY

Analysis of Adeno-associated Virus Progenitor Cell Transduction in Mouse Lung

(2008) Xiaoming Liu et al.   MOLECULAR THERAPY

Transduction Efficiencies of Novel AAV Vectors in Mouse Airway Epithelium In Vivo and Human Ciliated Airway Epithelium In Vitro

(2008) Maria P Limberis et al.   MOLECULAR THERAPY

CpG-free plasmids confer reduced inflammation and sustained pulmonary gene expression

(2008) Stephen C Hyde et al.   NATURE BIOTECHNOLOGY

Stem cells and cell therapies for cystic fibrosis and other lung diseases

(2008) Daniel J. Weiss   PULMONARY PHARMACOLOGY & THERAPEUTICS