Paul J. Orchard

TL;DR: Improvements in total body bone mineral content and growth velocity were associated with increases in growth velocity and reduced frequencies of bone fracture, indicating the feasibility of allogeneic bone marrow transplantation in the treatment of osteogenesis imperfecta and perhaps other mesenchymal stem cell disorders as well.

TL;DR: It is suggested that haploidentical NK cells can persist and expand in vivo and may have a role in the treatment of selected malignancies used alone or as an adjunct to HCT.

TL;DR: There is a high probability of survival in recipients of UCB grafts that are disparate in no more than 2 human leukocyte antigens when the grafts contain at least 1.7 x 10(5) CD34(+) cells per kilogram of recipient's body weight, and graft selection should be based principally on CD34 cell dose when multiple UCB units exist with an HLA disparity of 2 or less.

TL;DR: It is shown that TCIRG1, encoding the osteoclast-specific 116-kD subunit of the vacuolar proton pump, is mutated in five of nine patients with a diagnosis of infantile malignant osteopetrosis, indicating that mutations in TC IRG1 are a frequent cause of autosomal recessive osteopeterosis in humans.

TL;DR: Clinical responses of the first children to undergo allogeneic bone marrow transplantation (BMT) for severe osteogenesis imperfecta, a genetic disorder characterized by defective type I collagen, osteopenia, bone fragility, severe bony deformities, and growth retardation, are described.