Therapeutic Applications of Zinc Finger Nucleases
Zinc Finger Nuclease technology has been widely explored for therapeutic gene editing. Its ability to permanently modify DNA makes it particularly suited for treating inherited genetic disorders.
One prominent application involves editing immune cells to enhance their therapeutic potential. ZFNs have been used to modify T cells and stem cells, enabling long-lasting genetic changes that improve disease resistance or correct underlying defects.
In monogenic disorders, ZFNs can target and repair defective genes at their source. This approach addresses the root cause of disease rather than managing symptoms, offering the potential for long-term benefit.
Safety and precision are critical in therapeutic settings. ZFNs have demonstrated strong target specificity, reducing unintended genetic changes when properly designed and validated.
As gene-based therapies continue to advance, ZFN technology remains an important option for achieving durable genetic correction.