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Gene And Cell Therapy For Inborn Errors Of Metabolism

Cary O. Harding
Published 2017 · Medicine
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Shortcomings in contemporary therapy for inborn errors of metabolism (IEM) support the need for the development of novel treatment modalities. Cell therapy and gene therapy offer the promise of permanent cures for these rare disorders. The fundamental bases for both therapeutic approaches along with the therapeutic challenges are outlined in this chapter. For either technique, success lays in the ability to stably and safely provide sufficient and physiologically relevant numbers of non-disease cells in a target tissue to significantly affect the disease phenotype. The experimental rationale for these treatment approaches has been proven in animal models, and this review will focus upon clinical attempts at cell and gene therapy in human patients. Recent clinical trial successes in hemophilia, inherited immunodeficiency, genetic retinopathies, lipoprotein lipase (LPL) deficiency, the cerebral degenerative form of X-linked adrenoleukodystrophy, and metachromatic leukodystrophy illustrate the future promise of cell and gene therapy in the treatment of IEM.
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