Programmatic change: lung disease research in the era of induced pluripotency.

Human lung research has made remarkable progress over the last century largely through the use of animal models of disease. The challenge for the future is to translate these findings into human disease and bring about meaningful disease modification or even cure. The ability to generate transformative therapies in the future will require human tissue, currently scarce under the best of circumstances. Unfortunately, patient-derived somatic cells are often poorly characterized and have a limited life span in culture. Moreover, these cells are frequently obtained from patients with end-stage disease exposed to multiple drug therapies, leaving researchers with questions about whether their findings recapitulate disease-initiating processes or are simply the result of pharmacological intervention or subsequent host responses. The goal of studying early disease in multiple cell and tissue types has driven interest in the use of induced pluripotent stem cells (iPSCs) to model lung disease. These cells provide an alternative model for relevant lung research and hold promise in particular for studying the initiation of disease processes in genetic conditions such as heritable pulmonary arterial hypertension as well as other lung diseases. In this Perspective, we focus on potential iPSC use in pulmonary vascular disease research as a model for iPSC use in many types of advanced lung disease.

[1]  M. Humbert,et al.  Pulmonary arterial hypertension , 2013, Orphanet Journal of Rare Diseases.

[2]  T. Luedde,et al.  Mesenchymal Stem Cells Restore Lung Function by Recruiting Resident and Nonresident Proteins , 2011, Cell transplantation.

[3]  Li Li,et al.  MicroRNA-mediated conversion of human fibroblasts to neurons , 2011, Nature.

[4]  L. Hui,et al.  Induction of functional hepatocyte-like cells from mouse fibroblasts by defined factors , 2011, Nature.

[5]  Sayaka Sekiya,et al.  Direct conversion of mouse fibroblasts to hepatocyte-like cells by defined factors , 2011, Nature.

[6]  S. Yamanaka,et al.  The Use of Induced Pluripotent Stem Cells in Drug Development , 2011, Clinical pharmacology and therapeutics.

[7]  Jun S. Song,et al.  Incomplete DNA methylation underlies a transcriptional memory of somatic cells in human iPS cells , 2011, Nature Cell Biology.

[8]  Lior Gepstein,et al.  Modelling the long QT syndrome with induced pluripotent stem cells , 2011, Nature.

[9]  D. Roop,et al.  Osteoblasts Derived from Induced Pluripotent Stem Cells form Calcified Structures in Scaffolds Both In Vitro and In Vivo , 2011, Stem cells.

[10]  Fred H. Gage,et al.  A Model for Neural Development and Treatment of Rett Syndrome Using Human Induced Pluripotent Stem Cells , 2010, Cell.

[11]  Karl-Ludwig Laugwitz,et al.  Patient-specific induced pluripotent stem-cell models for long-QT syndrome. , 2010, New England Journal of Medicine.

[12]  R. Lafyatis,et al.  Generation of Transgene‐Free Lung Disease‐Specific Human Induced Pluripotent Stem Cells Using a Single Excisable Lentiviral Stem Cell Cassette , 2010, Stem cells.

[13]  V. Vedantham,et al.  Direct Reprogramming of Fibroblasts into Functional Cardiomyocytes by Defined Factors , 2010, Cell.

[14]  Christian Schuetz,et al.  Regeneration and orthotopic transplantation of a bioartificial lung , 2010, Nature Medicine.

[15]  Zhen W. Zhuang,et al.  Tissue-Engineered Lungs for in Vivo Implantation , 2010, Science.

[16]  K. Hochedlinger,et al.  Cell type of origin influences the molecular and functional properties of mouse induced pluripotent stem cells , 2010, Nature Biotechnology.

[17]  M. Longaker,et al.  Human iPS cell-based therapy: Considerations before clinical applications , 2010, Cell cycle.

[18]  Thomas Vierbuchen,et al.  Direct conversion of fibroblasts to functional neurons by defined factors , 2010, Nature.

[19]  M. Toshner,et al.  Endothelial progenitor cells in pulmonary hypertension – dawn of cell‐based therapy? , 2010, International journal of clinical practice. Supplement.

[20]  Krishanu Saha,et al.  Technical challenges in using human induced pluripotent stem cells to model disease. , 2009, Cell stem cell.

[21]  Robin Goland,et al.  Generation of pluripotent stem cells from patients with type 1 diabetes , 2009, Proceedings of the National Academy of Sciences.

[22]  Alan Colman,et al.  Pluripotent stem cells and disease modeling. , 2009, Cell stem cell.

[23]  A. Viale,et al.  Modeling Pathogenesis and Treatment of Familial Dysautonomia using Patient Specific iPSCs , 2009, Nature.

[24]  Horst Olschewski,et al.  Updated clinical classification of pulmonary hypertension. , 2009, Journal of the American College of Cardiology.

[25]  M. Humbert,et al.  Future perspectives for the treatment of pulmonary arterial hypertension. , 2009, Journal of the American College of Cardiology.

[26]  W. Chung,et al.  [Genetics and genomics of pulmonary arterial hypertension]. , 2014, Turk Kardiyoloji Dernegi arsivi : Turk Kardiyoloji Derneginin yayin organidir.

[27]  N. Weissmann,et al.  Cellular and molecular basis of pulmonary arterial hypertension. , 2009, Journal of the American College of Cardiology.

[28]  David J. Mooney,et al.  Growth Factors, Matrices, and Forces Combine and Control Stem Cells , 2009, Science.

[29]  Anne Lindgren,et al.  Directed Differentiation of Human‐Induced Pluripotent Stem Cells Generates Active Motor Neurons , 2009, Stem cells.

[30]  Shinya Yamanaka,et al.  Broader implications of defining standards for the pluripotency of iPSCs. , 2009, Cell stem cell.

[31]  George Q. Daley,et al.  Disease-Specific Induced Pluripotent Stem Cells , 2008, Cell.

[32]  Hynek Wichterle,et al.  Induced Pluripotent Stem Cells Generated from Patients with ALS Can Be Differentiated into Motor Neurons , 2008, Science.

[33]  R. Peterson,et al.  Dorsomorphin, a Selective Small Molecule Inhibitor of BMP Signaling, Promotes Cardiomyogenesis in Embryonic Stem Cells , 2008, PloS one.

[34]  M. Humbert,et al.  Clinical outcomes of pulmonary arterial hypertension in carriers of BMPR2 mutation. , 2008, American journal of respiratory and critical care medicine.

[35]  J. Knowles,et al.  Clinical implications of determining BMPR2 mutation status in a large cohort of children and adults with pulmonary arterial hypertension. , 2008, The Journal of heart and lung transplantation : the official publication of the International Society for Heart Transplantation.

[36]  Eric D. Adler,et al.  Human cardiovascular progenitor cells develop from a KDR+ embryonic-stem-cell-derived population , 2008, Nature.

[37]  B. Kerem,et al.  Consensus on the Use and Interpretation of Cystic Fibrosis Mutation Analysis in Clinical Practice , 2022 .

[38]  Gordon Keller,et al.  Differentiation of Embryonic Stem Cells to Clinically Relevant Populations: Lessons from Embryonic Development , 2008, Cell.

[39]  Shulan Tian,et al.  Induced Pluripotent Stem Cell Lines Derived from Human Somatic Cells , 2007, Science.

[40]  T. Ichisaka,et al.  Induction of Pluripotent Stem Cells from Adult Human Fibroblasts by Defined Factors , 2007, Cell.

[41]  T. Graf Faculty Opinions recommendation of Induction of pluripotent stem cells from adult human fibroblasts by defined factors. , 2007 .

[42]  Qiang Zhao,et al.  Effect of prepro-calcitonin gene-related peptide-expressing endothelial progenitor cells on pulmonary hypertension. , 2007, The Annals of thoracic surgery.

[43]  A. Moorman,et al.  Role of bone morphogenetic proteins in cardiac differentiation. , 2007, Cardiovascular research.

[44]  S. N. Murthy,et al.  Intratracheal mesenchymal stem cell administration attenuates monocrotaline-induced pulmonary hypertension and endothelial dysfunction. , 2007, American journal of physiology. Heart and circulatory physiology.

[45]  S. Kattman,et al.  Multipotent flk-1+ cardiovascular progenitor cells give rise to the cardiomyocyte, endothelial, and vascular smooth muscle lineages. , 2006, Developmental cell.

[46]  S. Yamanaka,et al.  Induction of Pluripotent Stem Cells from Mouse Embryonic and Adult Fibroblast Cultures by Defined Factors , 2006, Cell.

[47]  S. Mieno,et al.  Implantation of Mesenchymal Stem Cells Overexpressing Endothelial Nitric Oxide Synthase Improves Right Ventricular Impairments Caused by Pulmonary Hypertension , 2006, Circulation.

[48]  J. Loyd,et al.  The genetic approach in pulmonary fibrosis: can it provide clues to this complex disease? , 2006, Proceedings of the American Thoracic Society.

[49]  N. Rudarakanchana,et al.  Serotonin Increases Susceptibility to Pulmonary Hypertension in BMPR2-Deficient Mice , 2006, Circulation research.

[50]  R. Jaenisch,et al.  Generation of nuclear transfer-derived pluripotent ES cells from cloned Cdx2-deficient blastocysts , 2006, Nature.

[51]  D. Stewart,et al.  Rescue of Monocrotaline-Induced Pulmonary Arterial Hypertension Using Bone Marrow–Derived Endothelial-Like Progenitor Cells: Efficacy of Combined Cell and eNOS Gene Therapy in Established Disease , 2005, Circulation research.

[52]  Robert A Pearce,et al.  Specification of motoneurons from human embryonic stem cells , 2005, Nature Biotechnology.

[53]  J. Knowles,et al.  BMPR2 mutations in pulmonary arterial hypertension with congenital heart disease , 2004, European Respiratory Journal.

[54]  V. Tabar,et al.  Derivation of midbrain dopamine neurons from human embryonic stem cells. , 2004, Proceedings of the National Academy of Sciences of the United States of America.

[55]  M. Humbert,et al.  Pathologic assessment of vasculopathies in pulmonary hypertension. , 2004, Journal of the American College of Cardiology.

[56]  S. Hodge,et al.  BMPR2 germline mutations in pulmonary hypertension associated with fenfluramine derivatives , 2002, European Respiratory Journal.

[57]  R. Trembath,et al.  Primary Pulmonary Hypertension Is Associated With Reduced Pulmonary Vascular Expression of Type II Bone Morphogenetic Protein Receptor , 2002, Circulation.

[58]  W. Cookson,et al.  Genetics of asthma and allergic disease. , 2000, Human molecular genetics.

[59]  D. Stewart,et al.  Cell-based gene transfer to the pulmonary vasculature: Endothelial nitric oxide synthase overexpression inhibits monocrotaline-induced pulmonary hypertension. , 1999, American journal of respiratory cell and molecular biology.

[60]  J. Thomson,et al.  Embryonic stem cell lines derived from human blastocysts. , 1998, Science.

[61]  M. Kennedy,et al.  A common precursor for hematopoietic and endothelial cells. , 1998, Development.

[62]  D. Buff Primary pulmonary hypertension. , 1987, Annals of internal medicine.

[63]  M. Kaufman,et al.  Establishment in culture of pluripotential cells from mouse embryos , 1981, Nature.

[64]  関谷 明香,et al.  Direct conversion of mouse fibroblasts to hepatocyte-like cells by defined factors , 2012 .

[65]  N. Lee,et al.  Diagnosis and assessment of pulmonary arterial hypertension , 2010 .

[66]  I. Klimanskaya,et al.  Human embryonic stem cell lines derived from single blastomeres , 2006, Nature.