Faculty Research 1990 - 1999
Syngeneic bone marrow transplantation reduces the hearing loss associated with murine mucopolysaccharidosis type VII.
Document Type
Article
Publication Date
1995
Keywords
Bone-Marrow-Transplantation, Brain-Stem: ph, pp, pa, Disease-Models-Animal, Ear: pa, Evoked-Potentials-Auditory, Female, Glucuronidase: df, me, Hearing-Loss-Partial: pc, pa, Heterozygote, Homozygote, Human, Male, Mice, Mice-Mutant-Strains, Mucopolysaccharidosis-VII: pp, pa, th, SUPPORT-U-S-GOVT-NON-P-H-S, SUPPORT-U-S-GOVT-P-H-S, Transplantation-Isogeneic
First Page
2033
Last Page
2040
JAX Source
Blood 1995 Sep 1;86(5):2033-40
Grant
DK08546/DK/NIDDK, DK41082/DK/NIDDK, DC00048/DC/NIDCD
Abstract
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.
Recommended Citation
Sands MS,
Erway LC,
Vogler C,
Sly WS,
Birkenmeier EH.
Syngeneic bone marrow transplantation reduces the hearing loss associated with murine mucopolysaccharidosis type VII. Blood 1995 Sep 1;86(5):2033-40