Allogeneic stem cell transplantation for genetic disorders.
Thalassemia is the only genetic condition for which stem cell transplantation (SCT) has been considered the standard of care for achieving cure. With the advances in SCT, many new disorders have been added to the list of genetic diseases amenable to transplantation, especially the lysosomal storage disorders. The timing of the transplant is crucial in these conditions as the results are better when it is done before the onset of severe neuro-cognitive damage. Early diagnosis and referral, expedited donor searches, and newer techniques of SCT will improve the future results. This is especially important in the current era of expanded newborn screens now available in many states, including Kentucky.
Soni
Sandeep
S
Bone Marrow Transplantation University of Louisville, Kosair Childrens Hospital, KY 40202, USA.
D002675:Child, Preschool; D030342:Genetic Diseases, Inborn / Q000628:therapy*; D006453:Hemoglobinopathies / Q000628:therapy; D006801:Humans; D007223:Infant; D008297:Male; D008661:Metabolism, Inborn Errors / Q000628:therapy; D008059:Mucopolysaccharidosis I / Q000628:therapy*; D018579:Patient Selection; D033581:Stem Cell Transplantation; D014184:Transplantation, Homologous