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Lysosomal storage diseases associated with early-onset pathological changes may require prenatal therapy to avert the profound effects of the metabolic error on organs, especially the central nervous system. The present investigation determined the extent of expression of beta-mannosidase deficiency in the caprine fetus at 62 days of gestation, near the end of the period of immunotolerance when donor cells can engraft in various organs without immune rejection and supply missing enzyme. Three pairs of obligate carrier goats from the beta-mannosidosis colony were mated. Out of six fetuses delivered at 62 days of gestation, one (V385) was identified by measurement of beta-mannosidase activity as the only fetus affected with beta-mannosidosis. Thin-layer chromatography and quantitation of oligosaccharides revealed the presence of tri- and disaccharides, typical of beta-mannosidosis, only in V385. Morphological analysis revealed cytoplasmic vacuolation typical of beta-mannosidosis in V385; in thyroid, spinal cord, and kidney, the pattern of vacuolation was similar to, but less severe than, that observed previously in newborn affected goats. On the basis of these results, it will be possible to determine the effects of prenatal cell transplantation therapeutic strategies performed during the period of immunotolerance by monitoring phenotypic characteristics after treatment.
K L Lovell, and
F Matsuura, and
J Patterson, and
G Baeverfjord, and
N K Ames, and
M Z Jones
January 1984,
Journal of inherited metabolic disease,
K L Lovell, and
F Matsuura, and
J Patterson, and
G Baeverfjord, and
N K Ames, and
M Z Jones
January 1982,
Progress in clinical and biological research,
K L Lovell, and
F Matsuura, and
J Patterson, and
G Baeverfjord, and
N K Ames, and
M Z Jones
March 1986,
The Veterinary record,
K L Lovell, and
F Matsuura, and
J Patterson, and
G Baeverfjord, and
N K Ames, and
M Z Jones
January 1994,
Molecular and chemical neuropathology,
K L Lovell, and
F Matsuura, and
J Patterson, and
G Baeverfjord, and
N K Ames, and
M Z Jones
September 1989,
Veterinary pathology,
K L Lovell, and
F Matsuura, and
J Patterson, and
G Baeverfjord, and
N K Ames, and
M Z Jones
November 1988,
Veterinary pathology,
K L Lovell, and
F Matsuura, and
J Patterson, and
G Baeverfjord, and
N K Ames, and
M Z Jones
January 1983,
Advances in oto-rhino-laryngology,
K L Lovell, and
F Matsuura, and
J Patterson, and
G Baeverfjord, and
N K Ames, and
M Z Jones
May 1983,
Journal of neuropathology and experimental neurology,
K L Lovell, and
F Matsuura, and
J Patterson, and
G Baeverfjord, and
N K Ames, and
M Z Jones
May 1981,
The Journal of biological chemistry,
K L Lovell, and
F Matsuura, and
J Patterson, and
G Baeverfjord, and
N K Ames, and
M Z Jones
January 1991,
Journal of inherited metabolic disease,
