The application of bone marrow transplantation to the treatment of genetic diseases.

Genetic diseases can be treated by transplantation of either normal allogeneic bone marrow or, potentially, autologous bone marrow into which the normal gene has been inserted in vitro (gene therapy). Histocompatible allogeneic bone marrow transplantation is used for the treatment of genetic diseases whose clinical expression is restricted to lymphoid or hematopoietic cells. The therapeutic role of bone marrow transplantation in the treatment of generalized genetic diseases, especially those affecting the central nervous system, is under investigation. The response of a generalized genetic disease to allogeneic bone marrow transplantation may be predicted by experiments in vitro. Gene therapy can be used only when the gene responsible for the disease has been characterized. Success of gene therapy for a specific genetic disease may be predicted by its clinical response to allogeneic bone marrow transplantation.

[1]  A. Pearson,et al.  Bone marrow transplantation for glycogen storage disease type II (Pompé's disease) , 1986, The New England journal of medicine.

[2]  C. Caskey,et al.  Expression of human HPRT in the central nervous system of transgenic mice , 1985, Nature.

[3]  M. Nuwer,et al.  BONE-MARROW TRANSPLANTATION FOR METACHROMATIC LEUCODYSTROPHY , 1985, The Lancet.

[4]  G. Lucarelli,et al.  MARROW TRANSPLANTATION FOR THALASSAEMIA FOLLOWING BUSULPHAN AND CYCLOPHOSPHAMIDE , 1985, The Lancet.

[5]  A. Dilella,et al.  Gene transfer and expression of human phenylalanine hydroxylase. , 1985, Science.

[6]  R. Hammer,et al.  Novel developmental specificity in the nervous system of transgenic animals expressing growth hormone fusion genes , 1985, Nature.

[7]  D. Snover,et al.  Bone-marrow transplantation in the Maroteaux-Lamy syndrome (mucopolysaccharidosis type VI). Biochemical and clinical status 24 months after transplantation. , 1984, The New England journal of medicine.

[8]  R. Geha,et al.  Busulfan and total body irradiation as antihematopoietic stem cell agents in the preparation of patients with congenital bone marrow disorders for allogenic bone marrow transplantation. , 1984, Blood.

[9]  A. Look,et al.  Bone-marrow transplantation in a patient with sickle-cell anemia. , 1984, The New England journal of medicine.

[10]  G. Lucarelli,et al.  Allogeneic marrow transplantation for thalassemia. , 1984, Experimental hematology.

[11]  I. Verma,et al.  Expression of a retrovirus encoding human HPRT in mice. , 1984, Science.

[12]  E. Conde,et al.  Complete recovery of hemopoiesis following bone marrow transplant in a patient with unresponsive congenital hypoplastic anemia (Blackfan-Diamond syndrome). , 1984, Blood.

[13]  E. Ginns,et al.  Bone-marrow transplantation in severe Gaucher's disease. , 1984, The New England journal of medicine.

[14]  R. Dinsmore,et al.  Marrow transplantation for congenital disorders. , 1984, Seminars in hematology.

[15]  R C Willis,et al.  Partial phenotypic correction of human Lesch-Nyhan (hypoxanthine-guanine phosphoribosyltransferase-deficient) lymphoblasts with a transmissible retroviral vector. , 1984, The Journal of biological chemistry.

[16]  F. Scaravilli,et al.  Enzyme replacement in grafted nerve of twitcher mouse , 1983, Nature.

[17]  K. Sullivan,et al.  The pathophysiology and treatment of graft-versus-host disease. , 1983, Clinics in haematology.

[18]  R. Goldblum,et al.  Allogeneic bone marrow transplantation for chronic granulomatous disease. , 1982, The Journal of pediatrics.

[19]  A. Nicoll,et al.  HEALTH INSURANCE IN BRITAIN , 1982, The Lancet.

[20]  S. Perrine,et al.  SURFACE PROTEIN ABNORMALITIES IN LYMPHOCYTES AND PLATELETS FROM PATIENTS WITH WISKOTT-ALDRICH SYNDROME , 1981, The Lancet.

[21]  Jerry R. Hobbs,et al.  REVERSAL OF CLINICAL FEATURES OF HURLER'S DISEASE AND BIOCHEMICAL IMPROVEMENT AFTER TREATMENT BY BONE-MARROW TRANSPLANTATION , 1981, The Lancet.

[22]  R. Good,et al.  TRANSPLANTATION FOR ACUTE LEUKAEMIA WITH HLA-A AND B NONIDENTICAL PARENTAL MARROW CELLS FRACTIONATED WITH SOYBEAN AGGLUTININ AND SHEEP RED BLOOD CELLS , 1981, The Lancet.

[23]  R. Chaganti,et al.  Marrow transplantation for juvenile osteopetrosis. , 1981, The American journal of medicine.

[24]  R. Chaganti,et al.  Reconstitution of normal megakaryocytopoiesis and immunologic functions in Wiskott-Aldrich syndrome by marrow transplantation following myeloablation and immunosuppression with busulfan and cyclophosphamide , 1981 .

[25]  S. Feig,et al.  Prevention of graft rejection following bone marrow transplantation , 1981 .

[26]  P. Newburger,et al.  Correction of infantile agranulocytosis (Kostmann's syndrome) by allogeneic bone marrow transplantation. , 1980, The American journal of medicine.

[27]  S. Teitelbaum,et al.  Successful bone-marrow transplantation for infantile malignant osteopetrosis. , 1980, The New England journal of medicine.

[28]  D. Nathan,et al.  Successful use of multiagent immunosuppression in the bone marrow transplantation of sensitized patients. , 1978, Blood.

[29]  J. Belli,et al.  Complete correction of the Wiskott-Aldrich syndrome by allogeneic bone-marrow transplantation. , 1978, The New England journal of medicine.

[30]  G. Santos,et al.  BONE MARROW TRANSPLANTATION IN THE BUSULFAN‐TREATED RAT: III. RELATIONSHIP BETWEEN MYELOSUPPRESSION AND IMMUNOSUPPRESSION FOR CONDITIONING BONE MARROW RECIPIENTS , 1977, Transplantation.

[31]  J. G. Humble,et al.  Successful bone marrow transplant for Fanconi's anaemia. , 1977, British medical journal.

[32]  A. Garvin,et al.  The transport of lysosomal enzymes. , 1977, Journal of supramolecular structure.

[33]  Clift,et al.  Aplastic anemia treated by allogeneic bone marrow transplantation: a report on 49 new cases from Seattle. , 1976, Blood.

[34]  C. W. Parker,et al.  Drug allergy (first of three parts). , 1975, The New England journal of medicine.

[35]  E. Gelfand,et al.  Severe combined immunodeficiency and adenosine deaminase deficiency. , 1973, The New England journal of medicine.

[36]  R. Brady,et al.  Replacement therapy for inherited enzyme deficiency. Use of purified glucocerebrosidase in Gaucher's disease. , 1974, The New England journal of medicine.

[37]  J. Githens,et al.  Improved platelet function following bone marrow transplantation in an infant with the Wiskott-Aldrich syndrome. , 1973, The Journal of pediatrics.

[38]  E. Stiehm,et al.  Immunologic reconstitution in severe combined immunodeficiency wihout bone-marrow chromosomal chimerism. , 1972, The New England journal of medicine.

[39]  B. Nichols,et al.  Induced degradation of glycosaminoglycans in Hurler's and Hunter's syndromes by plasma infusion. , 1971, Proceedings of the National Academy of Sciences of the United States of America.

[40]  J. Seegmiller,et al.  Hypoxanthine-Guanine Phosphoribosyltransferase Deficiency in Gout , 1969 .

[41]  R. Gatti,et al.  Immunological reconstitution of sex-linked lymphopenic immunological deficiency. , 1968, Lancet.

[42]  R. Albertini,et al.  Bone-marrow transplantation in a patient with the Wiskott-Aldrich syndrome. , 1968, Lancet.