Gene transfer approaches to the lysosomal storage disorders

• Published in: Neurochemical research Vol. 24; no. 4; pp. 601 - 615
• Main Authors: BARRANGER, J. A, RICE, E. O, SWANEY, W. P
• Format: Journal Article
• Language: English
• Published: New York, NY Springer 01.04.1999; Springer Nature B.V
• Subjects: Animals; Biological and medical sciences; Biotechnology; Clinical Trials as Topic; Disease Models, Animal; Fundamental and applied biological sciences. Psychology; Gaucher Disease - therapy; Gene therapy; Gene Transfer Techniques; Glucosylceramidase - genetics; Health. Pharmaceutical industry; Hematopoietic Stem Cells - physiology; Humans; Industrial applications and implications. Economical aspects; Lysosomal Storage Diseases - therapy; Mice - genetics; Storage; Gene; Animal; Hematopoietic cell; Models; Lysosomal storage disease; Gene therapy; In vitro; Genetic transfer
• ISSN: 0364-3190; 1573-6903
• DOI: 10.1023/A:1022548232735

The work summarized in this paper used animal and cell culture models systems to develop gene therapy approaches for the lysosomal storage disorders. The results have provided the scientific basis for a clinical trial of gene transfer to hematopoietic stem cells (HSC) in Gaucher disease which is now in progress. The clinical experiment is providing evidence of HSC transduction, competitive engraftment of genetically corrected HSC, expression of the GC transgene, and the suggestion of a clinical response. In this paper we will review the progress made in Gaucher disease and include how gene transfer might be studied in other lysosomal storage disorders.