Table 3.

Results of breeding neonatally transplanted mucopolysaccharidosis VII adults as compared to mice receiving β-glucuronidase enzyme

Treatment of
breeding pairs
Number of
matings
Mean number
born (± SD)
Mean number
weaned (± SD)
% Survival
Normal 40 6.3 ± 2.2 5.9 ± 2.2 93.7% 
6xERT 7.0 ± 2.0 5.6 ± 1.5 80.0% 
3xBMT 6.7 ± 2.5 5.8 ± 2.8 86.6% 
Treatment of
breeding pairs
Number of
matings
Mean number
born (± SD)
Mean number
weaned (± SD)
% Survival
Normal 40 6.3 ± 2.2 5.9 ± 2.2 93.7% 
6xERT 7.0 ± 2.0 5.6 ± 1.5 80.0% 
3xBMT 6.7 ± 2.5 5.8 ± 2.8 86.6% 

Male and female MPS VII neonates were treated with either 6 weekly injections of recombinant murine GUS enzyme replacement therapy (6xERT) or 3 daily injections of +/+ syngeneic bone marrow without myeloablation (3xBMT).

ERT indicates enzyme replacement therapy; for other abbreviations see Tables 1 and 2.

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