Recombinant human α-glucosidase from rabbit milk in Pompe patients

Lancet

Volumn 356, Issue 9227, 2000, Pages 397-398

  Van Hout, Hannerieke Den ^a   Reuser, Arnold J J ^a   Vulto, Arnold G ^b   Christa B Loonen, M ^c   Cromme Dijkhuis, Adri ^d   Van Der Ploeg, Ans T ^e  

^a SOPHIA CHILDREN S HOSPITAL   (Netherlands)

^b Division of Pédiatrie Cardiology

^c Department of Child Neurology ^*

^d ERASMUS MEDICAL CENTER   (Netherlands)

^e ERASMUS UNIVERSITY ROTTERDAM   (Netherlands)

Author keywords

[No Author keywords available]

Indexed keywords

ALPHA GLUCOSIDASE;

CARDIOMYOPATHY; CASE REPORT; DRUG TOLERABILITY; ENZYME ACTIVITY; GLYCOGEN STORAGE DISEASE TYPE 2; HEART LEFT VENTRICLE MASS; HUMAN; INFANT; LETTER; MILK; PRIORITY JOURNAL; RABBIT;

EID: 0034729963     PISSN: 01406736     EISSN: None     Source Type: Journal    
DOI: 10.1016/s0140-6736(00)02533-2     Document Type: Letter

References (4)

1. Hirschhorn R. Glycogen storage disease type II: acid a-glucosidase (acid maltase) deficiency. In: Scriver CR, Beaudet AL, Sly WS, Valle D, eds. The metabolic and molecular bases of inherited disease, vol 1. New York: McGraw-Hill, 1995: 2443-64.
2. Fuller M, Van der Ploeg AT, Reuser AJJ, Anson DS, Hopwood JJ. Isolation and characterisation of a recombinant precursor form of lysosomal acid a-glucosidase. Eur J Biochem 1995; 234: 903-09.
3. Van Hove JLK, Yang HW, Wu J-Y, Brady RO, Chen YT. High level production of recombinant human lysosomal acid α-glucosidase in Chinese hamster ovary cells which targets to heart muscle and corrects glycogen accumulation in fibroblasts from patients with Pompe disease. Proc Nad Acad -Sa USA 1996; 93: 65-70.
4. Bijvoet AGA, Van Hirtum H, Kroos MA, et al. Human acid α-glucosidase from rabbit milk has therapeutic effect in mice with glycogen storage disease type II. Hum Molec Genet 1999; 8: 2145-53.