Prospects for enzyme replacement therapy in heritable metabolic disorders
| Autor: | P. G. Pentchev, R. O. Brady, F. S. Furbish, Andrew E. Gal, J. A. Barranger |
|---|---|
| Rok vydání: | 1982 |
| Předmět: |
chemistry.chemical_classification
medicine.medical_specialty Messenger RNA business.industry Allosteric regulation Enzyme replacement therapy Pharmacology medicine.disease Fabry disease Organ transplantation Metachromatic leukodystrophy chemistry.chemical_compound Enzyme chemistry medicine business DNA |
| Zdroj: | Inborn Errors of Metabolism in Humans ISBN: 9789400973275 |
| DOI: | 10.1007/978-94-009-7325-1_10 |
| Popis: | Since the discovery that insufficient activity of lipid catabolizing enzymes formed the basis of heritable lipid-storage disorders, a number of therapeutic strategies have been suggested1. Approaches to the treatment of such disorders included: (1) enzyme supplementation by implantation of normal or hybridized (corrected) patient cells; (2) organ transplantation; (3) administration of purified enzymes; (4) activation of catalytically defective mutated enzymes by allosteric agents; and (5) administration of appropriate messenger RNA or DNA cistrons2. |
| Databáze: | OpenAIRE |
| Externí odkaz: |
|