Precision Gene Editing With Zinc Finger Protein Technology

“The zinc fingers are using specifically designed versions of what our cells are already doing to regulate gene expression in the brain. We pair these ZFPs with the functional protein in order to create ZFP transcription factors. This functional domain could be anything; it could be something to upregulate or downregulate gene expression. We’re using this approach to target a wide array of neurological diseases.”

Among recent gene editing innovations is Sangamo Therapeutics’s zinc finger protein technology (ZFP), several abstracts on which were presented at the 24th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), May 12-15, 2021.¹

ZFPs are designed to be able to target genome sequences with single-nucleotide precision, high efficiency, and high specificity to limit off-target effects.² Preclinical research presented at ASGCT showed the potential of AAV-ZFP-transcription factors as a therapeutic approach in Parkinson disease, with evidence of downregulated alpha-synuclein mRNA expression in the brain.¹

GeneTherapyLive spoke with Amy Pooler, PhD, vice president of neuroscience at Sangamo Therapeutics, to learn more about the potential of ZFPs in treating a variety of central nervous system disorders. She also discussed the advantages of the technology over other gene editing strategies.

REFERENCES

1. Hatami A, Nguyen H, Roth T, et al. Alpha-synuclein repression using zinc finger protein transcription factors - A novel therapeutic approach for the treatment of Parkinson’s disease. Presented at: 2021 American Society of Gene & Cell Therapy Annual Meeting; May 12-15, 2021. Abstract 539.
2. Zinc Finger Platform. Sangamo Therapeutics. 2021. https://www.sangamo.com/technology/zinc-finger-platform