Genetic correction of induced pluripotent stem cells mediated by transcription activator-like effector nucleases targeting ALPL recovers enzyme activity and calcification in vitro.

[1]  D. Lo Furno,et al.  Functional role of mesenchymal stem cells in the treatment of chronic neurodegenerative diseases , 2018, Journal of cellular physiology.

[2]  Michel Sadelain,et al.  Gene therapy comes of age , 2018, Science.

[3]  E. Inada,et al.  Alkaline phosphatase and OCT‐3/4 as useful markers for predicting susceptibility of human deciduous teeth‐derived dental pulp cells to reprogramming factor‐induced iPS cells , 2017, Journal of investigative and clinical dentistry.

[4]  N. Bishop,et al.  Monitoring guidance for patients with hypophosphatasia treated with asfotase alfa. , 2017, Molecular genetics and metabolism.

[5]  A. Kurisaki,et al.  Simple and effective generation of transgene-free induced pluripotent stem cells using an auto-erasable Sendai virus vector responding to microRNA-302. , 2017, Stem cell research.

[6]  T. Ogata,et al.  Safety and efficacy of treatment with asfotase alfa in patients with hypophosphatasia: Results from a Japanese clinical trial , 2017, Clinical endocrinology.

[7]  M. Whyte Hypophosphatasia: Enzyme Replacement Therapy Brings New Opportunities and New Challenges , 2017, Journal of bone and mineral research : the official journal of the American Society for Bone and Mineral Research.

[8]  J. Riancho,et al.  Molecular and clinical analysis of ALPL in a cohort of patients with suspicion of Hypophosphatasia , 2017, American journal of medical genetics. Part A.

[9]  R. Herrmann,et al.  Mesenchymal Stromal Cell Therapy for Chronic Lung Allograft Dysfunction: Results of a First‐in‐Man Study , 2017, Stem cells translational medicine.

[10]  Anurag K Singh,et al.  Allogeneic Stem Cell Transplantation: A Historical and Scientific Overview. , 2016, Cancer research.

[11]  C. Dunbar,et al.  Gene Editing of Human Hematopoietic Stem and Progenitor Cells: Promise and Potential Hurdles. , 2016, Human gene therapy.

[12]  Y. Beguin,et al.  Clinical-scale expansion of mesenchymal stromal cells: a large banking experience , 2016, Journal of Translational Medicine.

[13]  T. Toki,et al.  Systematic Cellular Disease Models Reveal Synergistic Interaction of Trisomy 21 and GATA1 Mutations in Hematopoietic Abnormalities. , 2016, Cell reports.

[14]  M. Whyte Hypophosphatasia — aetiology, nosology, pathogenesis, diagnosis and treatment , 2016, Nature Reviews Endocrinology.

[15]  N. Bishop,et al.  Transformative therapy in hypophosphatasia , 2016, Archives of Disease in Childhood.

[16]  K. Ozono,et al.  Lethal hypophosphatasia successfully treated with enzyme replacement from day 1 after birth , 2016, European Journal of Pediatrics.

[17]  S. Yamanaka,et al.  From Genomics to Gene Therapy: Induced Pluripotent Stem Cells Meet Genome Editing. , 2015, Annual review of genetics.

[18]  J. Millán,et al.  Alkaline Phosphatase and Hypophosphatasia , 2015, Calcified Tissue International.

[19]  J. Millán,et al.  Prevention of Lethal Murine Hypophosphatasia by Neonatal Ex Vivo Gene Therapy Using Lentivirally Transduced Bone Marrow Cells. , 2015, Human gene therapy.

[20]  T. Taketani,et al.  Ex Vivo Expanded Allogeneic Mesenchymal Stem Cells with Bone Marrow Transplantation Improved Osteogenesis in Infants with Severe Hypophosphatasia , 2015, Cell transplantation.

[21]  Y. Matsumoto,et al.  Derivation of Mesenchymal Stromal Cells from Pluripotent Stem Cells through a Neural Crest Lineage using Small Molecule Compounds with Defined Media , 2014, PloS one.

[22]  Feng Chen,et al.  Targeted gene correction minimally impacts whole-genome mutational load in human-disease-specific induced pluripotent stem cell clones. , 2014, Cell stem cell.

[23]  Erin L. Doyle,et al.  TAL Effector Specificity for base 0 of the DNA Target Is Altered in a Complex, Effector- and Assay-Dependent Manner by Substitutions for the Tryptophan in Cryptic Repeat –1 , 2013, PloS one.

[24]  T. Taketani,et al.  Clinical and genetic aspects of hypophosphatasia in Japanese patients , 2013, Archives of Disease in Childhood.

[25]  Colby G Starker,et al.  In vivo Genome Editing Using High Efficiency TALENs , 2012, Nature.

[26]  Nick Bishop,et al.  Enzyme-replacement therapy in life-threatening hypophosphatasia. , 2012, The New England journal of medicine.

[27]  K. Ozono,et al.  Hypophosphatasia now draws more attention of both clinicians and researchers: A Commentary on prevelance of c. 1559delT in ALPL, a common mutation resulting in the perinatal (lethal) form of hypophosphatasias in Japanese and effects of the mutation on heterozygous carriers , 2011, Journal of Human Genetics.

[28]  S. Ikegawa,et al.  Prevalence of c.1559delT in ALPL, a common mutation resulting in the perinatal (lethal) form of hypophosphatasia in Japanese and effects of the mutation on heterozygous carriers , 2011, Journal of Human Genetics.

[29]  Xiaodong Chen,et al.  Mesenchymal stem cells: a new strategy for immunosuppression and tissue repair , 2010, Cell Research.

[30]  E. Mornet Hypophosphatasia , 2007, Orphanet journal of rare diseases.

[31]  T. Uchihashi,et al.  Common mutations F310L and T1559del in the tissue-nonspecific alkaline phosphatase gene are related to distinct phenotypes in Japanese patients with hypophosphatasia , 2005, European Journal of Pediatrics.

[32]  P. Schneider,et al.  Asp361Val Mutant of alkaline phosphatase found in patients with dominantly inherited hypophosphatasia inhibits the activity of the wild-type enzyme. , 2000, The Journal of clinical endocrinology and metabolism.

[33]  Peter J. Donovan,et al.  Derivation of pluripotent stem cells from cultured human primordial germ cells , 1998 .

[34]  Philippe Soriano,et al.  Tissue non-specific alkaline phosphatase is expressed in both embryonic and extraembryonic lineages during mouse embryogenesis but is not required for migration of primordial germ cells. , 1995, Development.

[35]  N. Bishop,et al.  Asfotase Alfa Treatment Improves Survival for Perinatal and Infantile Hypophosphatasia. , 2016, The Journal of clinical endocrinology and metabolism.