Animal Models of Alport Syndrome: Advancing the Prospects for Effective Human Gene Therapy
- 14 January 2000
- journal article
- review article
- Published by S. Karger AG in Nephron Experimental Nephrology
- Vol. 8 (1) , 1-7
- https://doi.org/10.1159/000020641
Abstract
Several animal models for Alport syndrome have been described. These are available for studies on the pathogenetic mechanisms of the disease, as well as for the development of new technologies for gene therapy in this progressive hereditary kidney disease. This review summarizes current knowledge on the molecular basis of Alport syndrome, and on the animal models which all remarkably well resemble the human disease. Recent work aimed at the development of gene therapy, including hurdles and progress are discussed.Keywords
This publication has 5 references indexed in Scilit:
- Glomerular Basement MembraneJournal of Biological Chemistry, 1998
- Molecular and functional defects in kidneys of mice lacking collagen alpha 3(IV): implications for Alport syndrome.The Journal of cell biology, 1996
- Coordinate Gene Expression of the α3, α4, and α5 Chains of Collagen Type IV: EVIDENCE FROM A CANINE MODEL OF X-LINKED NEPHRITIS WITH A COL4A5 GENE MUTATIONPublished by Elsevier ,1996
- Collagen IV alpha 3, alpha 4, and alpha 5 chains in rodent basal laminae: sequence, distribution, association with laminins, and developmental switches.The Journal of cell biology, 1994
- Identification of mutations in the α3(IV) and α4(IV) collagen genes in autosomal recessive Alport syndromeNature Genetics, 1994