Targeting CNS Disorders With Precision Gene Editing


The head of neuroscience at Sangamo Therapeutics discussed advances in understanding disease biology that allow for more specific gene targeting.

“The whole field of genomic medicine is really starting to open up. I think this is thanks to both the improved science and understanding of disease biology and also the availability of these new genomic medicine techniques. It's a really exciting time for neuroscience, because we may be able to finally intervene in some of these debilitating diseases in a way that could potentially deliver curative therapies.”

Sangamo Therapeutics is working to develop several new gene editing techniques, including their zinc finger protein (ZFP) technology in use in multiple central nervous system (CNS) disorders. Data from preclinical studies using ZFPs were presented at the 24th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), May 12-15, 2021.1

Sangamo cites the single-nucleotide precision, high efficiency, and high-specificity that ZFPs are designed to achieve that will limit off-target effects while effectively targeting genome sequences.2 Preclinical research has shown the 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 neuroscience research landscape and advances that are being made with gene editing and disease biology. She discussed several avenues the company is exploring with the ZFP platform and partnerships to advance research the company is involved in.

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.
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