Osteopetrosis
J Tolar, SL Teitelbaum, PJ Orchard - New England Journal of …, 2004 - Mass Medical Soc
This review of the mechanism of osteopetrosis incriminates molecular defects in osteoclasts
as the cause of the imbalance between bone formation by osteoblasts and bone resorption …
as the cause of the imbalance between bone formation by osteoblasts and bone resorption …
Diagnosis and management of osteopetrosis: consensus guidelines from the osteopetrosis working group
…, KL Insogna, MA Levine, PJ Orchard… - The Journal of …, 2017 - academic.oup.com
Background Osteopetrosis encompasses a group of rare metabolic bone diseases characterized
by impaired osteoclast activity or development, resulting in high bone mineral density. …
by impaired osteoclast activity or development, resulting in high bone mineral density. …
Defects in TCIRG1 subunit of the vacuolar proton pump are responsible for a subset of human autosomal recessive osteopetrosis
Osteopetrosis includes a group of inherited diseases in which inadequate bone resorption
is caused by osteoclast dysfunction. Although molecular defects have been described for …
is caused by osteoclast dysfunction. Although molecular defects have been described for …
Transplantability and therapeutic effects of bone marrow-derived mesenchymal cells in children with osteogenesis imperfecta
…, PL Gordon, M Neel, M Sussman, P Orchard… - Nature medicine, 1999 - nature.com
In principle, transplantation of mesenchymal progenitor cells would attenuate or possibly
correct genetic disorders of bone, cartilage and muscle, but clinical support for this concept is …
correct genetic disorders of bone, cartilage and muscle, but clinical support for this concept is …
Successful adoptive transfer and in vivo expansion of human haploidentical NK cells in patients with cancer
We previously demonstrated that autologous natural killer (NK)–cell therapy after
hematopoietic cell transplantation (HCT) is safe but does not provide an antitumor effect. We …
hematopoietic cell transplantation (HCT) is safe but does not provide an antitumor effect. We …
Hematopoietic cell therapy for metabolic disease
The lysosome, an intracellular organelle responsible for the intracellular sorting, recycling,
and digestion of organic molecules, was first described 50 years ago by Christian de Duve. 1 …
and digestion of organic molecules, was first described 50 years ago by Christian de Duve. 1 …
Transplantation of unrelated donor umbilical cord blood in 102 patients with malignant and nonmalignant diseases: influence of CD34 cell dose and HLA disparity on …
…, W Krivit, ML MacMillan, PJ Orchard… - Blood, The Journal …, 2002 - ashpublications.org
The potential benefits of unrelated donor marrow transplantation are offset by the immunologic
complications of graft-versus-host disease (GVHD) and infection. Therefore, we used …
complications of graft-versus-host disease (GVHD) and infection. Therefore, we used …
[BOOK][B] A right to flee
P Orchard - 2014 - books.google.com
… Drawing on extensive archival and secondary materials, Phil Orchard examines the interplay
among governments, individuals, and international organizations that has shaped how …
among governments, individuals, and international organizations that has shaped how …
Clinical responses to bone marrow transplantation in children with severe osteogenesis imperfecta
…, MD Neel, ME McCarville, PJ Orchard… - Blood, The Journal …, 2001 - ashpublications.org
… Arrows indicate the times of transplantation for patients (P). … Arrows indicate the times
of transplantation for patients (P). … Arrows indicate the times of transplantation for patients (P). …
of transplantation for patients (P). … Arrows indicate the times of transplantation for patients (P). …
[HTML][HTML] Hematopoietic stem-cell gene therapy for cerebral adrenoleukodystrophy
…, C Duncan, PL Musolino, PJ Orchard… - … England Journal of …, 2017 - Mass Medical Soc
Background In X-linked adrenoleukodystrophy, mutations in ABCD1 lead to loss of function
of the ALD protein. Cerebral adrenoleukodystrophy is characterized by demyelination and …
of the ALD protein. Cerebral adrenoleukodystrophy is characterized by demyelination and …