The potential to manipulate DNA sequences and insert genes with the use of zinc finger nucleases (ZFNs) has huge implications for human genetic disease therapeutics. One exciting example is a clinical trial that is using ZFN technology to disrupt the gene for the HIV co-receptor CCR5 and protect against the progression of HIV to AIDS. As shown in the infographic below, ZFNs introduce DNA sequence changes into individual genes to create deletions, insertions, or base substitutions. ZFN technology is a powerful tool that enables scientists to study the effects of these changes on gene function and may one day be used to treat human genetic disorders.
Maier D.A., Brennan A.L., Jiang S., Binder-Scholl G.K., Lee G., Plesa G., Zheng Z., Cotte J., Carpenito C., Wood T. & Spratt S.K. (2013). Efficient Clinical Scale Gene Modification via Zinc Finger Nuclease–Targeted Disruption of the HIV Co-receptor CCR5, Human Gene Therapy, 24 (3) 245-258. DOI: 10.1089/hum.2012.172
Introduction by Jillian L. Shaw.
Infographic by Jooyeun Lee.