Bone Marrow Transplantation for Genetic Diseases

Abstract

To the Editor: Dr. Barranger's comments in the Dec. 20 issue¹ omitted important aspects of the original proposal² that displacement bone marrow transplantation (DBMT) might correct at least 53 fatal genetic diseases. As of December 1984, it was known that 34 of those diseases have been satisfactorily corrected by DBMT (unpublished data). Dr. Barranger's neurologic caveats may apply to another nine diseases in which DBMT is being used (unpublished data), but his omissions offer alternative caveats: Avoidance of immunologic reactions against the component. When given to deficient animals, the normal component (usually a protein) can evoke antibodies impairing delivery, shortening . . .