Gene Therapy

KEY MESSSAGE:Will gene therapy one day become the ultimate application of genetic technology? Gene Therapy, the newest booklet in the Special Topics in Biology Series, addresses this question by exploring gene therapy as a complex technology for delivering therapeutic genes as a way to treat and cure human genetic diseases. Author Mary Colavito provides an overview of the basic science involved in gene therapy methods and chronicles the history of gene therapy by discussing successful and ongoing gene therapy treatments as well as adverse outcomes in some cases of gene therapy. Also discussed are challenges that must be overcome for gene therapy to become a more reliable and readily accessible approach for treating a multitude of genetic diseases that affect humans.Gene Therapy: Methods Currently Employed, Partial Successes and Ongoing Trials, Setbacks, Challenges, Future Approaches For all readers interested in gene therapy as a complex technology for delivering therapeutic genes as a way to treat and cure human genetic diseases.

Mary C. Colavito is a Professor in the Life Sciences Department at Santa Monica College in southern California.

Realizing the Promise of Gene Therapy: Successes, Setbacks, and Challenges	p. 1
Gene Therapy Methods	p. 3
Identifying a Disease That Is Likely to Respond to Gene Therapy	p. 3
Isolating a Functional Copy of the Gene	p. 8
Incorporating the Gene into a Carrier for Gene Delivery	p. 10
Determining Whether the Gene Product Is Made	p. 15
Partial Successes and Ongoing Trials	p. 16
Gene Therapy for Severe Combined Immune Deficiency (ADA-SCID)	p. 16
Gene Therapy for Cystic Fibrosis	p. 17
Gene Therapy for Canavan Disease	p. 18
Setbacks	p. 20
Immune Reactions Targeting Cells Carrying the Vector	p. 20
Inadvertent Activation of Cancer-Causing Genes by a Vector	p. 20
Insufficient Numbers of Cells Producing the Gene Product	p. 22
Challenges	p. 22
Safe and Effective Delivery of Genes	p. 23
Production of a Sufficient Amount of Gene Product	p. 30
Achieving a Lasting Improvement for the Patient	p. 31
Future Approaches	p. 31
Correction of a Patient's DNA	p. 31
Interfering with the Production of the Gene Product	p. 33
Treating Complex Conditions Resulting from Multiple Gene Defects	p. 38
Conclusion	p. 39
Resources for Students and Educators	p. 39
For Students	p. 39
For Educators	p. 41
Table of Contents provided by Ingram. All Rights Reserved.

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Summary

Author Biography

Table of Contents

Supplemental Materials