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 …

Background Osteopetrosis encompasses a group of rare metabolic bone diseases characterized
by impaired osteoclast activity or development, resulting in high bone mineral density. …

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 …

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 …

Osteopetrosis includes a group of inherited diseases in which inadequate bone resorption
is caused by osteoclast dysfunction. Although molecular defects have been described for …

Preclinical models have shown that transplantation of marrow mesenchymal cells has the
potential to correct inherited disorders of bone, cartilage, and muscle. The report describes …

Mucopolysaccharidosis type I (MPS I) is a rare autosomal recessive inherited disease,
caused by deficiency of the enzyme α-L-iduronidase, resulting in accumulation of the …

Background In X-linked adrenoleukodystrophy, mutations in ABCD1 lead to loss of function
of the ALD protein. Cerebral adrenoleukodystrophy is characterized by demyelination and …

Approximately 20,000 hematopoietic cell transplantation (HCT) procedures are performed
in the United States annually. With advances in transplantation technology and supportive …

Cerebral X-linked adrenoleukodystrophy (X-ALD) is a disorder of very-long-chain fatty acid
metabolism, adrenal insufficiency, and cerebral demyelination. Death occurs within 2 to 5 …