Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells

Inclusion Criteria: * Lethal or sublethal genetic disease of blood cells, who lack a fully histocompatible sibling or other family donor * Genetic diseases that would be candidates for this protocol includes those that have been shown to benefit from allogeneic HSCT: Red blood cell defects, Leukocyte defects/ Primary immune deficiencies, Platelets defects, Metabolic/storage disorders and Stem cell defects. * Renal: creatinine clearance or glomerular filtration rate (GFR) ≥50 ml/min/1.73m2 and not requiring dialysis. * Pulmonary: FEV1, FVC and DLCO (corrected for hemoglobin) ≥ 50% predicted. if unable to perform pulmonary function tests, then O2 saturation ≥ 92% in room air. * Cardiac: Left ventricular ejection fraction at rest must be ≥ 40%, or shortening fraction ≥ 26% * Hepatic: Bilirubin ≤3x upper limit of normal (ULN) and ALT and AST ≤ 5x for age (with the exception of isolated hyperbilirubinemia due to Gilbert's syndrome). * Patients will be 0-21 years of age. * Disease specific inclusion criteria (as applicable per protocol). Exclusion Criteria: * Recipients should not have any of the general exclusion criteria, and disease specific exclusion criteria when applicable. * Patient with histocompatible sibling * End-organ failure that precludes the ability to tolerate the transplant procedure, including the conditioning regimen. * Creatinine clearance or GFR \< 50 ml/min/1.73m2 or renal failure requiring dialysis. * Congenital heart disease resulting in congestive heart …