2003
DOI: 10.1016/j.ymgme.2003.08.022
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Enzyme replacement therapy in the mouse model of Pompe disease

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Cited by 194 publications
(228 citation statements)
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“…Liver had the highest protein levels for both enzymes (Fig. 3e), which coincides with the fact that liver has the most abundant expression of M6PR that facilitates transportation of rhGAA or FabGAA to the lysosomes [30]. Quadriceps had the lowest levels for both rhGAA and FabGAA, which is consistent with the previously observed low efficacy of ERT in skeletal muscles [30].…”
Section: Distribution Of Fabgaa In Mouse Tissues Was Heterogeneoussupporting
confidence: 86%
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“…Liver had the highest protein levels for both enzymes (Fig. 3e), which coincides with the fact that liver has the most abundant expression of M6PR that facilitates transportation of rhGAA or FabGAA to the lysosomes [30]. Quadriceps had the lowest levels for both rhGAA and FabGAA, which is consistent with the previously observed low efficacy of ERT in skeletal muscles [30].…”
Section: Distribution Of Fabgaa In Mouse Tissues Was Heterogeneoussupporting
confidence: 86%
“…3e), which coincides with the fact that liver has the most abundant expression of M6PR that facilitates transportation of rhGAA or FabGAA to the lysosomes [30]. Quadriceps had the lowest levels for both rhGAA and FabGAA, which is consistent with the previously observed low efficacy of ERT in skeletal muscles [30]. Both rhGAA and FabGAA proteins presented predominately as the mature lysosomal forms (76 and 67 kDa) in all tissues, indicating that both proteins were destined to the lysosomes (Fig.…”
Section: Distribution Of Fabgaa In Mouse Tissues Was Heterogeneoussupporting
confidence: 85%
See 1 more Smart Citation
“…In fact, after intravenous administration of rhGAA in Pompe mice, the majority of the enzyme was taken up by liver, followed by heart and skeletal muscle [15]. A similar rhGAA uptake pattern was also observed in patients with Pompe disease (unpublished data).…”
Section: Discussionsupporting
confidence: 61%
“…Patients with the most severe infantile form normally die within the first year of life, while in the milder late-onset forms, muscle weakness is the primary symptom and respiratory failure is the major cause of significant morbidity and mortality [1]. Enzyme replacement therapy (ERT) with recombinant human GAA (rhGAA, alglucosidase alfa) is currently the only available therapy for Pompe disease with remarkable success, but also with significant limitations including the high cost for life-long administration of high-dose enzyme, poor clinical outcomes caused by the formation of high-titer anti-rhGAA antibodies in the majority of patients, and reduced efficacy in the skeletal muscle [2][3][4][5]. Thus, there is an unmet need to develop improved therapies for Pompe disease.…”
Section: Introductionmentioning
confidence: 99%