Targeting Glucocerebrosidase to Macrophages for Effective Treatment of Patients with Gaucher Disease: Setting the Paradigm of a “Fit for Purpose” Approach to Enzyme Replacement Therapy
Publisher
Springer New York
Reference26 articles.
1. Aghion A (1934) La maladie de Gaucher dans l’enfance. Thèse, Paris
2. Barton NW, Furbish FS, Murray GJ, Garfield M, Brady RO (1990) Therapeutic response to intravenous infusions of glucocerebrosidase in a patient with Gaucher disease. Proc Natl Acad Sci U S A 87:1913–1916
3. Barton NW, Brady RO, Dambrosia JM, DiBisceglie AM, Doppelt SH, Hill SC, Mankin HJ, Murray GJ, Parker RI, Argoff CE, Grewal RP, Yu K-T (1991) Replacement therapy for inherited enzyme deficiency—macrophage-targeted glucocerebrosidase for Gaucher’s disease. N Engl J Med 324:1464–1470
4. Brady RO (1966) The sphingolipidoses. N Engl J Med 275:312–318
5. Brady RO, Furbish FS (1982) Enzyme replacement therapy: specific targeting of exogenous enzymes to storage cells. In: Martonosi AN (ed) Membranes and transport, vol 2. Plenum, New York, pp 587–592