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Full Description
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. A list of web resources and relevant print publications are provided at the end of this Special Topics Booklet from Benjamin Cummings.
Contents
Realizing the Promise of Gene Therapy: Successes, Setbacks and Challenges
Methods Currently Employed
Identifying a disease that is likely to respond to gene therapy
Isolating a functional copy of the gene
Incorporating the gene into a carrier (vector)
Delivering the gene
Incorporating the Gene into a Chromosome
Determining whether the gene product is produced
Partial Successes and Ongoing Trials
Gene Therapy for Severe Combined Immune Deficiency (SCID)
Gene Therapy for Cystic Fibrosis
Gene Therapy for Canavan Disease
Setbacks
Insufficient Numbers of Cells Producing the Gene Product (Andrew Gobea's therapy for SCID)
Immune Reactions Targeting Cells Carrying the Vector (death of Jesse Gelsinger after therapy for Ornithine
Transcarbamylase Deficiency [OTC])
Inadvertent Activation of Cancer-Causing Genes by a Vector (development of leukemia in French patients treated for SCID)
Challenges
Safe and Effective Delivery of the Gene
Production of a sufficient amount of gene product
Achieving a lasting improvement for the patient
Future Approaches
Controlling the chromosomal region where the delivered gene is inserted
Targeting specific sites on chromosomes for gene integration
Preventing insertion near cancer-causing genes by using insulators
Alternate Delivery Methods to Avoid Complications with Vectors
Liposomes containing genes without the need for vectors
Nanoparticles packaging DNA molecules for entry through nuclear pores
Expanding the means for achieving the goals of gene therapy by exploring alternative ways to assist the cell in producing the functional gene product or blocking the formation of a harmful product
Correction of the patient's DNA
Regulating production of the gene product by methods that affect RNA
RNA interference leading to degradation of specific RNAs
Antisense RNA preventing protein production
Correction of RNA, through control of splicing
