NDLI: Gene targeting approaches to complex genetic diseases: atherosclerosis and essential hypertension.

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Gene targeting approaches to complex genetic diseases: atherosclerosis and essential hypertension.

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Gene targeting approaches to complex genetic diseases: atherosclerosis and essential hypertension.

Content Provider	World Health Organization (WHO)-Global Index Medicus
Author	Smithies, O. Maeda, N.
Description	Author Affiliation: Smithies O ( Department of Pathology, University of North Carolina at Chapel Hill 27599-7525, USA.);
Abstract	Gene targeting allows precise, predetermined changes to be made in a chosen gene in the mouse genome. To date, targeting has been used most often for generation of animals completely lacking the product of a gene of interest. The resulting 'knockout' mice have confirmed some hypotheses, have upset others, but have rarely been uninformative. Models of several human genetic diseases have been produced by targeting--including Gaucher disease, cystic fibrosis, and the fragile X syndrome. These diseases are primarily determined by defects in single genes, and their modes of inheritance are well understood. When the disease under study has a complex etiology with multiple genetic and environmental components, the generation of animal models becomes more difficult but no less valuable. The problems associated with dissecting out the individual genetic factors also increases substantially and the distinction between causation and correlation is often difficult. To prove causation in a complex system requires rigorous adherence to the principle that the experiments must allow detection of the effects of changing only a single variable at one time. Gene targeting experiments, when properly designed, can test the effects of a precise genetic change completely free from the effects of differences in any other genes (linked or unlinked to the test gene). They therefore allow proofs of causation.
ISSN	00278424
e-ISSN	10916490
Journal	Proceedings of the National Academy of Sciences of the United States of America
Issue Number	12
Volume Number	92
Language	English
Publisher	National Academy of Sciences
Publisher Date	1995-07-01
Publisher Place	United States
Access Restriction	Open
Subject Keyword	Arteriosclerosis Genetics Gene Targeting Hypertension Animals Disease Models, Animal Research Support, U.S. Gov't, P.H.S. Multidisciplinary
Content Type	Text
Resource Type	Article
Subject	Multidisciplinary

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