Abstract
As knowledge of the genes and molecular events involved in specific kidney disorders expands, so do the potential applications for treating the disorders on fundamental levels. Progress in gene therapy for genetic diseases for which molecular events are simpler or better characterized has already been substantial. In addition, exciting first steps in targeting genes specifically to the kidney have emerged, although much work in defining kidney-specific genes and gene regulatory elements still needs to be done. To provide a framework for understanding the concepts and problems involved in gene therapy, we will discuss basic aspects of gene structure and regulation, gene delivery vectors, steps necessary to achieve tissue- and cell-specific expression of delivered genes, and some present and future applications of gene therapy in kidney diseases.
| Original language | English (US) |
|---|---|
| Pages (from-to) | 57-69 |
| Number of pages | 13 |
| Journal | Seminars in Nephrology |
| Volume | 15 |
| Issue number | 1 |
| State | Published - 1995 |
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ASJC Scopus subject areas
- Nephrology
Cite this
The potential of gene therapy for treatment of kidney diseases. / Riley, D. J.; Lee, W. H.
In: Seminars in Nephrology, Vol. 15, No. 1, 1995, p. 57-69.Research output: Contribution to journal › Article
}
TY - JOUR
T1 - The potential of gene therapy for treatment of kidney diseases
AU - Riley, D. J.
AU - Lee, W. H.
PY - 1995
Y1 - 1995
N2 - As knowledge of the genes and molecular events involved in specific kidney disorders expands, so do the potential applications for treating the disorders on fundamental levels. Progress in gene therapy for genetic diseases for which molecular events are simpler or better characterized has already been substantial. In addition, exciting first steps in targeting genes specifically to the kidney have emerged, although much work in defining kidney-specific genes and gene regulatory elements still needs to be done. To provide a framework for understanding the concepts and problems involved in gene therapy, we will discuss basic aspects of gene structure and regulation, gene delivery vectors, steps necessary to achieve tissue- and cell-specific expression of delivered genes, and some present and future applications of gene therapy in kidney diseases.
AB - As knowledge of the genes and molecular events involved in specific kidney disorders expands, so do the potential applications for treating the disorders on fundamental levels. Progress in gene therapy for genetic diseases for which molecular events are simpler or better characterized has already been substantial. In addition, exciting first steps in targeting genes specifically to the kidney have emerged, although much work in defining kidney-specific genes and gene regulatory elements still needs to be done. To provide a framework for understanding the concepts and problems involved in gene therapy, we will discuss basic aspects of gene structure and regulation, gene delivery vectors, steps necessary to achieve tissue- and cell-specific expression of delivered genes, and some present and future applications of gene therapy in kidney diseases.
UR - http://www.scopus.com/inward/record.url?scp=0028821229&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=0028821229&partnerID=8YFLogxK
M3 - Article
C2 - 7538691
AN - SCOPUS:0028821229
VL - 15
SP - 57
EP - 69
JO - Seminars in Nephrology
JF - Seminars in Nephrology
SN - 0270-9295
IS - 1
ER -