scholarly journals Murine mucopolysaccharidosis type VII: long term therapeutic effects of enzyme replacement and enzyme replacement followed by bone marrow transplantation.

1997 ◽  
Vol 99 (7) ◽  
pp. 1596-1605 ◽  
Author(s):  
M S Sands ◽  
C Vogler ◽  
A Torrey ◽  
B Levy ◽  
B Gwynn ◽  
...  
Keyword(s):  
Bone Marrow ◽  
Bone Marrow Transplantation ◽  
Marrow Transplantation ◽  
Therapeutic Effects ◽  
Mucopolysaccharidosis Type ◽  
Enzyme Replacement

scholarly journals Syngeneic bone marrow transplantation reduces the hearing loss associated with murine mucopolysaccharidosis type VII

Blood ◽  
1995 ◽  
Vol 86 (5) ◽  
pp. 2033-2040 ◽  
Author(s):  
MS Sands ◽  
LC Erway ◽  
C Vogler ◽  
WS Sly ◽  
EH Birkenmeier
Keyword(s):  
Bone Marrow ◽  
Hearing Loss ◽  
Bone Marrow Transplantation ◽  
Marrow Transplantation ◽  
Mucopolysaccharidosis Type ◽  
Lysosomal Storage ◽  
Middle Ear Mucosa ◽  
Mps Vii ◽  
The Impact

MPS VII mice are deficient in beta-glucuronidase and share many clinical, biochemical, and pathologic characteristics with human mucopolysaccharidosis type VII (MPS VII). We have shown that syngeneic bone marrow transplantation (BMT) prolongs survival and reduces lysosomal storage in many organs of the MPS VII mouse. In this report, we quantify the hearing loss and determine the impact of syngeneic BMT on the development of deafness and the associated pathology in the MPS VII mouse. Eleven weeks after syngeneic BMT performed at birth, treated MPS VII mice had normal auditory-evoked brainstem responses (ABR), whereas untreated MPS VII mice had ABR thresholds 43 dB higher than normal. Treated MPS VII mice had beta-glucuronidase-positive cells in the temporal bone and in the subepithelial connective tissue of the external auditory canal. There was less thickening of the tympanic membrane and middle ear mucosa and decreased distortion of the ossicles and the cochlear bone. Although transplanted MPS VII mice had increased ABR thresholds by 33 weeks of age, four of the six had thresholds 12 to 32 dB lower than untreated mutants. These data indicate that syngeneic BMT in newborn MPS VII mice prevents early hearing loss and, in some animals, results in long-term improved auditory function.

Evaluating the Patients with Thalassemia Major for Long-Term Endocrinological Complications After Bone Marrow Transplantation

2014 ◽  
Vol 31 (7) ◽  
pp. 616-623 ◽  
Author(s):  
Bilge Aldemir-Kocabaş ◽  
Gülsün Tezcan-Karasu ◽  
İffet Bircan ◽  
Oğuz Bircan ◽  
Anıl Aktaş-Samur ◽  
...  
Keyword(s):  
Bone Marrow ◽  
Bone Marrow Transplantation ◽  
Marrow Transplantation ◽  
Thalassemia Major
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