Zinc Finger Nuclease: A versatile emerging tool to diagnose human disease

Zinc-finger nucleases are synthetic restriction enzymes that cut DNA into more manageable pieces. It is a well-known technology in the field of genome editing, which is based on the DNA repair process. The vast range of applications of zinc finger nuclease, including target validation, functional genome editing, cell screening, and cell-based optimization, will make it the fundamental technology for biotechnology businesses in the future.

A sedentary lifestyle has increased the prevalence of chronic diseases, including cardiovascular disease, cancer, high blood pressure, obesity, and others. This has prompted excessive research and development to create novel therapeutic agents to treat different disease conditions. Benefits of Zinc Finger Nuclease (ZFN) include permanent and heritable mutations, effectiveness for some mammalian somatic cell types, ability to induce gene editing with a single transfection, and selection without the need for antibiotic screening. These advantages have made it easier for researchers to conduct their studies with fewer tools.

Potentially all of the issues with therapeutic gene delivery by viruses can be avoided with the ZFN-encoding plasmid-based method. Ex vivo treatment employing a patient's stem cells is probably one of the earliest therapeutic uses for ZFNs. The cells could be multiplied in vitro and reinserted into the patient after altering the stem cell genome to create differentiated cells with the desired activities. Initial targets will probably include the IL2R gene, the b-globin gene for gene correction, and the CCR5 gene for mutagenesis and disablement, as these are the root causes of monogenic illnesses.
It is employed in the clinical trial of CD4+ human T-cells for the treatment of AIDS as well as the manipulation of plants and animals for scientific purposes.
Craig Wilen from the University of Pennsylvania and his colleagues provided some initial information about gene therapy to prevent CD4 cell CXCR4. HIV may enter T-cells via both CCR5 and CXCR4; thus, blocking both would be necessary to protect a cell from infection properly. The repair mechanism causes mutations that prevent CD4 cells from expressing co-receptors.
This team also utilised Sangamo's zinc finger nuclease technology. It has been demonstrated that modifying mature CD4 T cells can protect from HIV infection, at least temporarily. Similar gene therapy techniques are currently being used on hematopoietic stem cells, which produce all subtypes of blood cells, including CD4 cells. It could be more difficult to suppress CXCR4 than CCR5.
The hepatitis B virus (HBV) kills millions yearly and chronically infects millions. Zinc-finger nucleases (ZFNs) cleave as dimers to break HBV DNA. However, it is still a relatively new development in diagnostics.