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Gene and Cell-Replacement Therapy in the Treatment of Type 1 Diabetes : How High Must the Standards Be Set?

Halban, Philippe A; Kahn, Steven E; Lernmark, Åke LU and Rhodes, Christopher J. (2001) In Diabetes 50(10). p.2181-2191
Abstract

Recent advances in molecular and cell biology may allow for the development of novel strategies for the treatment and cure of type 1 diabetes. In particular, it is now possible to envisage restoration of insulin secretion by gene or cell-replacement therapy. The β-cell is, however, remarkably sophisticated, and many of the features of this highly differentiated secretory cell will have to be faithfully mimicked in surrogate cells. In particular, insulin is normally secreted in a well-regulated fashion in rapid response to the metabolic needs of the individual and most specifically (but not exclusively) to changes in circulating levels of glucose. Such regulated secretion will be indispensable in order to avoid both hyper- and... (More)

Recent advances in molecular and cell biology may allow for the development of novel strategies for the treatment and cure of type 1 diabetes. In particular, it is now possible to envisage restoration of insulin secretion by gene or cell-replacement therapy. The β-cell is, however, remarkably sophisticated, and many of the features of this highly differentiated secretory cell will have to be faithfully mimicked in surrogate cells. In particular, insulin is normally secreted in a well-regulated fashion in rapid response to the metabolic needs of the individual and most specifically (but not exclusively) to changes in circulating levels of glucose. Such regulated secretion will be indispensable in order to avoid both hyper- and hypoglycemic episodes and depends on the ability of cells to store insulin in secretory granules before exocytosis in response to physiological stimuli. Furthermore, any newly created insulin-secreting cell will have to be able to adapt to alterations in insulin requirements that accompany changes with exercise, body weight, and aging. Fine tuning of insulin secretion over the longer term will also be important to avoid "clinical shifting" that could be caused by over-insulinization, including increased adiposity and cardiovascular disease. Finally, it will be necessary to ensure that newly created or implanted (surrogate) β-cells are protected in some way from recognition by the immune system and in particular from autoimmune destruction.

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author
publishing date
type
Contribution to journal
publication status
published
in
Diabetes
volume
50
issue
10
pages
11 pages
publisher
American Diabetes Association Inc.
external identifiers
  • scopus:0035491791
ISSN
0012-1797
language
English
LU publication?
no
id
9daa35a1-a842-4d30-ab28-69f80da586b9
date added to LUP
2017-09-07 09:36:29
date last changed
2018-01-07 12:18:16
@article{9daa35a1-a842-4d30-ab28-69f80da586b9,
  abstract     = {<p>Recent advances in molecular and cell biology may allow for the development of novel strategies for the treatment and cure of type 1 diabetes. In particular, it is now possible to envisage restoration of insulin secretion by gene or cell-replacement therapy. The β-cell is, however, remarkably sophisticated, and many of the features of this highly differentiated secretory cell will have to be faithfully mimicked in surrogate cells. In particular, insulin is normally secreted in a well-regulated fashion in rapid response to the metabolic needs of the individual and most specifically (but not exclusively) to changes in circulating levels of glucose. Such regulated secretion will be indispensable in order to avoid both hyper- and hypoglycemic episodes and depends on the ability of cells to store insulin in secretory granules before exocytosis in response to physiological stimuli. Furthermore, any newly created insulin-secreting cell will have to be able to adapt to alterations in insulin requirements that accompany changes with exercise, body weight, and aging. Fine tuning of insulin secretion over the longer term will also be important to avoid "clinical shifting" that could be caused by over-insulinization, including increased adiposity and cardiovascular disease. Finally, it will be necessary to ensure that newly created or implanted (surrogate) β-cells are protected in some way from recognition by the immune system and in particular from autoimmune destruction.</p>},
  author       = {Halban, Philippe A and Kahn, Steven E and Lernmark, Åke and Rhodes, Christopher J.},
  issn         = {0012-1797},
  language     = {eng},
  number       = {10},
  pages        = {2181--2191},
  publisher    = {American Diabetes Association Inc.},
  series       = {Diabetes},
  title        = {Gene and Cell-Replacement Therapy in the Treatment of Type 1 Diabetes : How High Must the Standards Be Set?},
  volume       = {50},
  year         = {2001},
}