English (United Kingdom)

https://curated-unify.zendy.io/wp-json/zendy-region/v1/featured_content/oa?rat=en

https://curated-unify.zendy.io/wp-json/zendy-region/v1/highlighted_journal/

Global: Zendy Plus annual

Presents the access of premium content as premium feature

Premium Content

Presents the keyphrase highlighting as premium feature

Keyphrase Highlighting

Presents the summarisation as premium feature

Summarisation

Insights

Presents the pdf analysis as premium feature

PDF Analysis

Presents the zaia usage as premium feature

ZAIA

Global: Zendy Plus monthly

Global: Zendy Tools annual

Global: Zendy Tools monthly

Global: Zendy Free Plan

The importance of gene therapy strategies for the treatment of malignancies is highlighted by the fact that there are (at the time of writing) 823 cancer gene therapy clinical trials world‐ wide that are actively, or have yet to begin recruiting patients (www.clinicaltrials.gov ac‐ cessed November 2012). The potential of the delivery of genetic material for therapeutic purposes has long been recognised, but to this point, has yet to be successfully translated. Strategies that have proved promising in the in vitro setting have stumbled when exposed to the complexities of the in vivo environment. Classically involving the delivery of plasmid DNA (pDNA) that encodes a therapeutic protein product, the field of gene therapy has evolved to encompass not only delivery of therapeutic DNA, but also micro(miRNA), short hairpin(shRNA) and small interfering RNAs (siRNA) and oligodeoxynucleotides (ODNs) [1]. Despite the evolution of the technology for altering the genotype of target cells and tis‐ sues, the problem of overcoming the biological barriers that limit the efficacies of these tech‐ nologies remains. These barriers exist at both systemic and local levels. To date, the only approved nucleic acid-based treatments for clinical use are an antisense ODN for the treat‐ ment of cytomegalovirus retinitis [2], and pegaptanib sodium (Macugen), an RNA aptamer targeted against VEGF-165 and used to treat age-related macular degeneration [3]. This chap‐ ter will focus on the biological barriers faced by non-viral vectors for gene therapy, strategies that have been employed to overcome these barriers, and will conclude by documenting the state of the art technologies being used to propel non-viral gene therapies forward.

Cancer Gene Therapy – Key Biological Concepts in the Design of Multifunctional Non-Viral Delivery Systems