TY - JOUR
T1 - Bone Marrow Transplantation for Genetic Diseases
AU - Krivit, William
AU - Whitley, Chester B.
PY - 1987/4/23
Y1 - 1987/4/23
N2 - Four recent editorials in the Journal have reviewed the potential for increasing the therapeutic armamentarium for genetic disease.1 2 3 4 Bone marrow transplantation has been considered in each of these editorials. Now, in this issue of the Journal, Lucarelli et al. present impressive data on the use of marrow transplantation for the treatment of β-thalassemia.5 In severely affected patients 8 to 15 years of age, functional engraftment as measured by correction of the hematologic abnormality has been produced. In the future, clinical success will be measured by continued freedom from hemolysis and the necessity of repeated transfusions, resulting in an eventual…
AB - Four recent editorials in the Journal have reviewed the potential for increasing the therapeutic armamentarium for genetic disease.1 2 3 4 Bone marrow transplantation has been considered in each of these editorials. Now, in this issue of the Journal, Lucarelli et al. present impressive data on the use of marrow transplantation for the treatment of β-thalassemia.5 In severely affected patients 8 to 15 years of age, functional engraftment as measured by correction of the hematologic abnormality has been produced. In the future, clinical success will be measured by continued freedom from hemolysis and the necessity of repeated transfusions, resulting in an eventual…
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U2 - 10.1056/NEJM198704233161710
DO - 10.1056/NEJM198704233161710
M3 - Editorial
C2 - 3550465
AN - SCOPUS:0023662843
SN - 0028-4793
VL - 316
SP - 1085
EP - 1087
JO - New England Journal of Medicine
JF - New England Journal of Medicine
IS - 17
ER -