Convection perfusion of glucocerebrosidase for neuronopathic Gaucher's disease

Annals of Neurology

Volumn 57, Issue 4, 2005, Pages 542-548

  Lonser, Russell R ^a   Walbridge, Stuart ^a   Murray, Gary J ^a   Aizenberg, Michele R ^a,b   Vortmeyer, Alexander O ^a   Aerts, Johannes M F G ^c   Brady, Roscoe O ^a   Oldfield, Edward H ^a  

^a NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE   (United States)

^b GEORGE WASHINGTON UNIVERSITY MEDICAL CENTER   (United States)

^c UNIVERSITY OF AMSTERDAM   (Netherlands)

Author keywords

[No Author keywords available]

Indexed keywords

IMIGLUCERASE;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; BLOOD BRAIN BARRIER; BRAIN CORTEX; BRAIN STEM; CENTRAL NERVOUS SYSTEM DISEASE; CONTROLLED STUDY; DRUG SAFETY; ENZYME ACTIVITY; ENZYME REPLACEMENT; FRONTAL LOBE; GAUCHER DISEASE; IMMUNOHISTOCHEMISTRY; NONHUMAN; NUCLEAR MAGNETIC RESONANCE IMAGING; PHENOTYPE; PONS; PRIMATE; PRIORITY JOURNAL; RAT; WHITE MATTER;

ANIMALS; BRAIN; CONVECTION; DRUG DELIVERY SYSTEMS; GAUCHER DISEASE; GLUCOSYLCERAMIDASE; IMMUNOHISTOCHEMISTRY; MACACA MULATTA; MAGNETIC RESONANCE IMAGING; MALE; PERFUSION; RATS;

EID: 16344372850     PISSN: 03645134     EISSN: None     Source Type: Journal    
DOI: 10.1002/ana.20444     Document Type: Article

References (24)

1. Brady RO, Kanfer JN, Bradley RM, Shapiro D. Demonstration of a deficiency of glucocerebroside-cleaving enzyme in Gaucher's disease. J Clin Invest 1966;45:1112-1115.
2. Verity MA, Montasir M. Infantile Gaucher's disease: neuropathology, acid hydrolase activities and negative staining observations. Neuropadiatrie 1977;8:89-100.
3. Barton NW, Brady RO, Dambrosia JM, et al. Replacement therapy for inherited enzyme deficiency-macrophage-targeted glucocerebrosidase for Gaucher's disease. N Engl J Med 1991; 324:1464-1470.
4. Brady RO, Pentchev PG, Gal AE, et al. Replacement therapy for inherited enzyme deficiency. Use of purified glucocerebrosidase in Gaucher's disease. N Engl J Med 1974;291:989-993.
5. Brady RO. Therapy for the sphingolipidoses. Arch Neurol 1998;55:1055-1056.
6. Altarescu G, Hill S, Wiggs E, et al. The efficacy of enzyme replacement therapy in patients with chronic neuronopathic Gaucher's disease. J Pediatr 2001;138:539-547.
7. Weinreb NJ, Charrow J, Andersson HC, et al. Effectiveness of enzyme replacement therapy in 1028 patients with type 1 Gaucher disease after 2 to 5 years of treatment: a report from the Gaucher Registry. Am J Med 2002;113:112-119.
8. Schiffmann R, Brady RO. New prospects for the treatment of lysosomal storage diseases. Drugs 2002;62:733-742.
9. Prows CA, Sanchez N, Daugherty C, Grabowski GA. Gaucher disease: enzyme therapy in the acute neuronopathic variant. Am J Med Genet 1997;71:16-21.
10. Bobo RH, Laske DW, Akbasak A, et al. Convection-enhanced delivery of macromolecules in the brain. Proc Natl Acad Sci U S A 1994;91:2076-2080.
11. Chen MY, Lonser RR, Morrison PF, et al. Variables affecting convection-enhanced delivery to the striatum: a systematic examination of rate of infusion, cannula size, infusate concentration, and tissue-cannula sealing time. J Neurosurg 1999;90: 315-320.
12. Bembi B, Ciana G, Zanatta M, et al. Cerebrospinal-fluid infusion of alglucerase in the treatment for acute neuronopathic Gaucher's disease. Pediatr Res 1995;38:A425.
13. Barranger JA, Rapoport SI, Fredericks WR, et al. Modification of the blood-brain barrier: increased concentration and fate of enzymes entering the brain. Proc Natl Acad Sci U S A 1979; 76:481-485.
14. Lonser RR, Gogate N, Morrison PF, et al. Direct convective delivery of macromolecules to the spinal cord. J Neurosurg 1998;89:616-622.
15. Lonser RR, Weil RJ, Morrison PF, et al. Direct convective delivery of macromolecules to peripheral nerves. J Neurosurg 1998;89:610-615.
16. Zirzow GC, Sanchez OA, Murray GJ, et al. Delivery, distribution, and neuronal uptake of exogenous mannose-terminal glucocerebrosidase in the intact rat brain. Neurochem Res 1999; 24:301-305.
17. Lonser RR, Walbridge S, Garmestani K, et al. Successful and safe perfusion of the primate brainstem: in vivo magnetic resonance imaging of macromolecular distribution during infusion. J Neurosurg 2002;97:905-913.
18. Laske DW, Morrison PF, Lieberman DM, et al. Chronic interstitial infusion of protein to primate brain: determination of drug distribution and clearance with single-photon emission computerized tomography imaging. J Neurosurg 1997;87: 586-594.
19. Laske DW, Youle RJ, Oldfield EH. Tumor regression with regional distribution of the targeted toxin TF-CRM107 in patients with malignant brain tumors. Nat Med 1997;3: 1362-1368.
20. Tybulewicz VL, Tremblay ML, LaMarca ME, et al. Animal model of Gaucher's disease from targeted disruption of the mouse glucocerebrosidase gene. Nature 1992;357:407-410.
21. Daniels LB, Coyle PJ, Glew RH, et al. Brain glucocerebrosidase in Gaucher's disease. Arch Neurol 1982;39:550-556.
22. Wenger DA, Roth S, Kudoh T, et al. Biochemical studies in a patient with subacute neuropathic Gaucher disease without visceral glucosylceramide storage. Pediatr Res 1983;17:344-348.
23. Schueler U, Kaneski C, Murray G, et al. Uptake of mannose-terminal glucocerebrosidase in cultured human cholinergic and dopaminergic neuron cell lines. Neurochem Res 2002;27: 325-330.
24. Burudi EM, Riese S, Stahl PD, Regnier-Vigoureux A. Identification and functional characterization of the mannose receptor in astrocytes. Glia 1999;25:44-55.