Transplantation increasingly is being used as treatment for children with end-stage organ diseases, hematopoietic rescue from therapy used to treat malignancies, and as cure for primary immune deficiencies. This article…
The armamentarium of biologic therapies targeting specific elements of the immune system is rapidly expanding. This review describes the spectrum of infectious complications associated to date with each of the…
Hematopoietic stem cell transplantation (HSCT) is a treatment for multiple medical conditions that result in bone marrow failure and as an antineoplastic adoptive immunotherapy for hematologic malignancies. HSCT is associated…
The concept of gene therapy emerged as a way of correcting monogenic inherited diseases by introducing a normal copy of the mutated gene into at least some of the patients’ cells. Although…
Replacement of disease-causing stem cells with healthy ones has been achieved clinically via hematopoietic cell transplantation (HCT) for the last 40 years, as a treatment modality for a variety of…
Since the early 1980s T-cell depletion has allowed haploidentical bone marrow transplantation to be performed in patients with primary immunodeficiency for whom a matched sibling donor was not available, without…
Severe combined immunodeficiency (SCID) is fatal in infancy unless corrected with allogeneic bone marrow transplants (BMT), preferably from a family-related genotypically HLA-identical donor (RID) or phenotypically HLA-matched family donor (PMD)….
Curative treatment of Severe Combined Immunodeficiency (SCID) by Hematopoietic Cell Transplantation (HCT) remains a challenge, in particular in infants presenting with serious, poorly controllable complications. In the absence of a…
It is now more than 40 years since the first successful allogeneic hematopoietic stem cell transplantation (HSCT) for a child with severe combined immunodeficiency (SCID). In the succeeding years, HSCT…
The last 40 years has seen the emergence of hematopoietic stem cell transplantation as a therapeutic modality for fatal diseases and as a curative option for individuals born with inherited…
