Gene Therapy in Oncology

Published on 04/03/2015 by admin

Filed under Hematology, Oncology and Palliative Medicine

Last modified 04/03/2015

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Gene Therapy in Oncology

James E. Talmadge and Kenneth H. Cowan

Summary of Key Points

Recent Advances in Gene Therapy

• Chimeric antigen receptor transduction of peripheral blood T lymphocytes for delivery of specifically cytotoxic immunoreactivity

• Silencing of gene expression with short hairpin RNA delivered with nanoparticle or lentiviral delivery vectors

• Regulated transgene expression

• Phase 3 studies ongoing

Ideal Vector Attributes

• Can be targeted either physically or via promoter expression and is nontoxic, noninflammatory, and nonimmunogenic

• Should have the potential to incorporate a large transgene and result in high levels of both transduction and transgene expression

• Duration of transgene expression and/or genomic integration ought to be regulatable

Current Concerns Regarding Gene Therapy

• Gene therapy using an adenovirus vector has been implicated in the death of at least one patient

• Leukemic transformation by insertional mutagenesis

Future Directions of Gene Therapy

• The choice of disease, clinical implementation, and vector are critically important to the future development of successful gene therapy

• Because of deficiencies in gene delivery and targeting, as well as expression levels, it is critical to pair protocols with specific vector attributes

Self-Assessment Questions

1. What toxicity issue has been observed with retroviral vectors?

A Integration into the host genome and development of leukemia

B Development of inflammatory responses to the vector

C Recombination and formation of a replication-competent virus

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