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9 - Gene Therapy and Imaging of Transgene Expression in Living Subjects

Published online by Cambridge University Press:  07 September 2010

Sanjiv Sam Gambhir  and
Shahriar S. Yaghoubi
By
Shahriar S. Yaghoubi  and
Sanjiv Sam Gambhir
Sanjiv Sam Gambhir
Affiliation:
Stanford University School of Medicine, California
Shahriar S. Yaghoubi
Affiliation:
Stanford University School of Medicine, California
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Summary

GENE THERAPY

Broadly defined, gene therapy is the process of delivering genetic materials into a patient or cells from the patient to modify the expression of one or more genes to achieve a therapeutic outcome for the patient. Hence, a transgene delivery procedure may be intended for the following reasons: (1) to initiate the expression of the transgene in specific tissues or the entire body of the patient, (2) to reduce the expression of specific endogenous genes at a target site or the entire body of the patient, and (3) to enhance the expression of specific endogenous genes in target tissues or the entire body of the patient. The success of a gene therapy procedure depends on several factors including (1) specific cell targeting when it is necessary to cause changes in gene expression only in specific cells of the patient's body and avoid modification of gene expression in all other cells. This may be necessary to avoid side effects and deliver sufficient gene expression modifying genetic material to the target cells; (2) efficiency of transgene delivery to achieve sufficient modification of gene expression in target cells; (3) modified gene expression for sufficient time duration; and (4) lack of immune reaction to the product of the therapeutic transgene, when immune reaction will reduce efficacy or potency of the therapeutic procedure. Investigators in the field of gene therapy are working hard to solve problems associated with all of these factors.

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Molecular Imaging with Reporter Genes , pp. 227 - 238
Publisher: Cambridge University Press
Print publication year: 2010

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