Hypophosphatasia

[1]  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.

[2]  T. Odrljin,et al.  Asfotase alfa therapy for children with hypophosphatasia. , 2016, JCI insight.

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

[4]  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.

[5]  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.

[6]  T. Taketani.  Neurological Symptoms of Hypophosphatasia. , 2015, Sub-cellular biochemistry.

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

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

[9]  D. Fauvert,et al.  A Molecular‐Based Estimation of the Prevalence of Hypophosphatasia in the European Population , 2011, Annals of human genetics.

[10]  J. Millán,et al.  Rescue of severe infantile hypophosphatasia mice by AAV-mediated sustained expression of soluble alkaline phosphatase. , 2011, Human gene therapy.

[11]  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.

[12]  T. Taketani,et al.  New bone formation by allogeneic mesenchymal stem cell transplantation in a patient with perinatal hypophosphatasia. , 2009, The Journal of pediatrics.

[13]  M. Kassem,et al.  The use of mesenchymal (skeletal) stem cells for treatment of degenerative diseases: Current status and future perspectives , 2009, Journal of cellular physiology.

[14]  R. Terkeltaub,et al.  Enzyme Replacement Therapy for Murine Hypophosphatasia , 2007, Journal of bone and mineral research : the official journal of the American Society for Bone and Mineral Research.

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

[16]  R. Cahill,et al.  Infantile hypophosphatasia: transplantation therapy trial using bone fragments and cultured osteoblasts. , 2007, The Journal of clinical endocrinology and metabolism.

[17]  W. Sly,et al.  Enhancement of drug delivery to bone: characterization of human tissue-nonspecific alkaline phosphatase tagged with an acidic oligopeptide. , 2006, Molecular genetics and metabolism.

[18]  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.

[19]  J. Kurtzberg,et al.  Marrow Cell Transplantation for Infantile Hypophosphatasia , 2003, Journal of bone and mineral research : the official journal of the American Society for Bone and Mineral Research.

[20]  Y. Masamune,et al.  Novel Drug Delivery System to Bone Using Acidic Oligopeptide: Pharmacokinetic Characteristics and Pharmacological Potential , 2001, Journal of drug targeting.