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Updated: Jun 5, 2026
09:11
Genome Editing with CompoZr Custom Zinc Finger Nucleases (ZFNs)
Published on: June 14, 2012
Zinc-finger nuclease based genome surgery: it's all about specificity
Eva-Maria Händel1, Toni Cathomen
1Hannover Medical School, Department of Experimental Hematology, Carl-Neuberg-Strasse 1, Hannover, Germany.
Current Gene Therapy
|December 25, 2010
Summary
Zinc-finger nucleases (ZFNs) enable precise genome engineering for gene therapy. These designer nucleases are advancing human therapeutics, with clinical trials underway for HIV treatment.
Area of Science:
- Molecular Biology
- Gene Therapy
- Genomics
Background:
- Therapeutic genome engineering is crucial for gene therapy.
- Recent advances allow targeted modification of complex genomes.
- Zinc-finger nucleases (ZFNs) are key tools in genome engineering.
Purpose of the Study:
- To review technological innovations in ZFN development.
- To provide an overview of the current state of ZFN technology.
- To discuss ZFN applications in human gene therapy.
Main Methods:
- ZFNs have been used to edit genomes in over 50 gene loci across 11 model organisms.
- ZFN-mediated genome surgery has been performed in primary human cells.
- Clinical trials are investigating ZFNs for HIV treatment by targeting the CCR5 receptor.
Main Results:
- ZFNs achieve high allelic frequencies in genome editing experiments.
- ZFN technology has advanced to enable targeted genome modification.
- ZFNs are being explored in human gene therapy, including clinical trials.
Conclusions:
- ZFNs are a powerful technology for genome engineering and gene therapy.
- Specificity and ZFN-associated toxicity are critical parameters for therapeutic applications.
- ZFNs hold significant promise for treating genetic diseases and viral infections like HIV.
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