Experimental Enzyme Replacement in Genetic and Other Disorders
- 1 September 1976
- journal article
- research article
- Published by Taylor & Francis in Hospital Practice
- Vol. 11 (9), 49-58
- https://doi.org/10.1080/21548331.1976.11706996
Abstract
To correct a genetic defect, it would not be enough, say, to inject the missing enzyme, since the body's immune defenses would rapidly destroy it. A “Trojan horse” is needed to evade immune surveillance. The evolution of such an approach is described, as well as how it has been used to “cure” Tay-Sachs disease in culture, utilizing immunoglobulin-disguised liposomes to bring hexosaminidase A to deficient cells.Keywords
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