Bone marrow transplantation for Sanfilippo disease type B

1992 ◽  
Vol 15 (6) ◽  
pp. 911-918 ◽  
Author(s):  
A. Vellodi ◽  
E. Young ◽  
M. New ◽  
C. Pot-Mees ◽  
K. Hugh-Jones
Keyword(s):  
Bone Marrow ◽  
Bone Marrow Transplantation ◽  
Marrow Transplantation ◽  
Disease Type ◽  
Type B ◽  
Sanfilippo Disease

scholarly journals Treatment of Niemann-Pick disease type B by allogeneic bone marrow transplantation.

BMJ ◽  
1987 ◽  
Vol 295 (6610) ◽  
pp. 1375-1376 ◽  
Author(s):  
A Vellodi ◽  
J R Hobbs ◽  
N M O'Donnell ◽  
B S Coulter ◽  
K Hugh-Jones
Keyword(s):  
Bone Marrow ◽  
Bone Marrow Transplantation ◽  
Marrow Transplantation ◽  
Allogeneic Bone Marrow Transplantation ◽  
Disease Type ◽  
Allogeneic Bone ◽  
Type B ◽  
Niemann Pick Disease ◽  
Niemann Pick ◽  
Pick Disease

Bone Marrow Transplantation in Glycogen Storage Disease Type 1b

2008 ◽  
Vol 152 (2) ◽  
pp. 286-288 ◽  
Author(s):  
Germaine Pierre ◽  
Geothy Chakupurakal ◽  
Patrick Mckiernan ◽  
Chris Hendriksz ◽  
Sarah Lawson ◽  
...  
Keyword(s):  
Bone Marrow ◽  
Bone Marrow Transplantation ◽  
Glycogen Storage Disease ◽  
Marrow Transplantation ◽  
Storage Disease ◽  
Glycogen Storage Disease Type ◽  
Glycogen Storage ◽  
Disease Type

scholarly journals Evidence for engraftment of donor-type multipotent CD34+ cells in a patient with selective T-lymphocyte reconstitution after bone marrow transplantation for B-SCID

Blood ◽  
1994 ◽  
Vol 84 (10) ◽  
pp. 3584-3589 ◽  
Author(s):  
GE Tjonnfjord ◽  
R Steen ◽  
OP Veiby ◽  
W Friedrich ◽  
T Egeland
Keyword(s):  
Bone Marrow ◽  
Bone Marrow Transplantation ◽  
T Lymphocyte ◽  
Marrow Transplantation ◽  
B Lymphocyte ◽  
B Lymphopoiesis ◽  
Type B ◽  
Cd34 Cells ◽  
Donor Type

Severe combined immunodeficiencies (SCID), a heterogeneous group of disorders of infancy, are fatal without treatment directed at immunologic reconstitution. Allogeneic bone marrow transplantation (BMT), which is such a treatment presents some unique features in SCID, especially when T-lymphocyte-depleted HLA haploidentical allografts are used. Donor-type T lymphopoiesis, less often B lymphopoiesis, develops, whereas myelopoiesis remains the recipient-type. Little is known about the engrafting cells in this peculiar lymphohematopoietic chimerism and the pathophysiology of the frequent failure of B-lymphocyte reconstitution. To address these issues, we purified CD34+ BM cells from a patient with selective T-lymphocyte reconstitution after HLA haploidentical BMT for B-SCID. Phenotypic analysis of CD34+ cells was performed by flow cytometry, and functional studies of donor- and recipient-type CD34+ cells were performed in vitro. Donor-type CD34+ cells, constituting approximately 2% of the CD34+ cells, were detected; both CD34+ HLA-DR- cells and CD34+ cells coexpressing B-(CD10 and CD19) and T-(CD2 and CD7) lymphocyte-associated cell surface molecules. Donor- type CD34+ cells coexpressing myeloid-associated molecules (CD13, CD14, CD15, and CD33) were undetectable. However, donor-type CD34+ myeloid progenitors could be shown in functional assays. Recipient-type CD34+ cells coexpressing B- and T-lymphocyte- as well as myeloid-associated molecules were detected, but recipient-type CD34+ cells could not be driven into T-lymphocyte differentiation in vitro. These findings provide evidence for engraftment of multipotent stem cells in our patient with B-SCID. Furthermore, the failure of B-lymphocyte reconstitution cannot be explained by lack of donor-type B-lymphocyte progenitors. Donor-type B lymphopoiesis and myelopoiesis are prevented by an unidentified mechanism.

scholarly journals Evidence for engraftment of donor-type multipotent CD34+ cells in a patient with selective T-lymphocyte reconstitution after bone marrow transplantation for B-SCID

Blood ◽  
1994 ◽  
Vol 84 (10) ◽  
pp. 3584-3589 ◽  
Author(s):  
GE Tjonnfjord ◽  
R Steen ◽  
OP Veiby ◽  
W Friedrich ◽  
T Egeland
Keyword(s):  
Bone Marrow ◽  
Bone Marrow Transplantation ◽  
T Lymphocyte ◽  
Marrow Transplantation ◽  
B Lymphocyte ◽  
B Lymphopoiesis ◽  
Type B ◽  
Cd34 Cells ◽  
Donor Type

Abstract Severe combined immunodeficiencies (SCID), a heterogeneous group of disorders of infancy, are fatal without treatment directed at immunologic reconstitution. Allogeneic bone marrow transplantation (BMT), which is such a treatment presents some unique features in SCID, especially when T-lymphocyte-depleted HLA haploidentical allografts are used. Donor-type T lymphopoiesis, less often B lymphopoiesis, develops, whereas myelopoiesis remains the recipient-type. Little is known about the engrafting cells in this peculiar lymphohematopoietic chimerism and the pathophysiology of the frequent failure of B-lymphocyte reconstitution. To address these issues, we purified CD34+ BM cells from a patient with selective T-lymphocyte reconstitution after HLA haploidentical BMT for B-SCID. Phenotypic analysis of CD34+ cells was performed by flow cytometry, and functional studies of donor- and recipient-type CD34+ cells were performed in vitro. Donor-type CD34+ cells, constituting approximately 2% of the CD34+ cells, were detected; both CD34+ HLA-DR- cells and CD34+ cells coexpressing B-(CD10 and CD19) and T-(CD2 and CD7) lymphocyte-associated cell surface molecules. Donor- type CD34+ cells coexpressing myeloid-associated molecules (CD13, CD14, CD15, and CD33) were undetectable. However, donor-type CD34+ myeloid progenitors could be shown in functional assays. Recipient-type CD34+ cells coexpressing B- and T-lymphocyte- as well as myeloid-associated molecules were detected, but recipient-type CD34+ cells could not be driven into T-lymphocyte differentiation in vitro. These findings provide evidence for engraftment of multipotent stem cells in our patient with B-SCID. Furthermore, the failure of B-lymphocyte reconstitution cannot be explained by lack of donor-type B-lymphocyte progenitors. Donor-type B lymphopoiesis and myelopoiesis are prevented by an unidentified mechanism.

scholarly journals Epstein-barr virus-positive recipient type B-cells survive in a “complete chimera” after allogeneic bone-marrow transplantation

1988 ◽  
Vol 42 (5) ◽  
pp. 672-676 ◽  
Author(s):  
Heinrich H. Gerhartz ◽  
Johann Mittermüller ◽  
Anand Raghavachar ◽  
Helga Schmetzer ◽  
Christof Clemmi ◽  
...  
Keyword(s):  
Bone Marrow ◽  
Bone Marrow Transplantation ◽  
B Cells ◽  
Marrow Transplantation ◽  
Epstein Barr Virus ◽  
Allogeneic Bone Marrow Transplantation ◽  
Allogeneic Bone ◽  
Type B ◽  
Barr Virus ◽  
Epstein Barr
Sign in / Sign up

Export Citation Format

Share Document