Oncology (Williston Park). 1992 Mar;6(3):51-8; discussion 58, 63-6.

Bone marrow transplantation for genetic disorders.

Source

Bone Marrow Transplant Program, Hackensack Medical Center, New Jersey.

Abstract

Allogeneic bone marrow transplantation has been successfully utilized to correct the lymphoid and/or hematopoietic abnormalities characterizing a wide array of lethal genetic disorders. Examples include severe combined immunodeficiency, Wiskott-Aldrich syndrome, thalassemia major, sickle cell anemia, and several types of lysosomal storage diseases. Most marrow transplant recipients require preparation with myeloablative doses of chemotherapy, with or without additional radiation therapy, to ensure engraftment of allogeneic marrow. This approach has dramatically changed the long-term outlook for many children with otherwise lethal disorders.

PMID:: 1533145; [PubMed - indexed for MEDLINE]

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[Bone marrow transplantation in the treatment of various genetically determined diseases]. [Pol Tyg Lek. 1992]
[Bone marrow transplantation in the treatment of various genetically determined diseases].
Robak T. Pol Tyg Lek. 1992 Mar 2-9; 47(9-10):207-10.
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Review High-dose therapy and bone marrow transplantation. [Semin Oncol. 1985]
Review High-dose therapy and bone marrow transplantation.
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Cited by 2 PubMed Central articles

Review Gene therapy for lysosomal storage diseases (LSDs) in large animal models. [ILAR J. 2009]
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