using zinc fingers to control genes
Every cell in our body contains the same genes. Yet, neurons, muscle cells, and skin cells look and work differently. To specialize, our cells turn certain genes on or off when needed. Our cells often regulate gene expression by relying on zinc finger proteins, a class of transcription factors that recognize and bind to specific sequences of DNA code, enabling a cell to differentiate the targeted gene among thousands of others. The family of zinc finger proteins (ZFPs) are among the most common genes in the human genome.
Sangamo is developing zinc finger protein transcription factors (ZFP-TFs) as a novel therapeutic approach for diseases of the central nervous system, including tauopathies, Huntington’s disease, and amyotrophic lateral sclerosis (ALS).
Advantages of ZFP TFs
Innovation in delivery promises to provide new opportunities for the potential use of in vivo genomic therapies in tissues outside the liver. We are investing in multiple delivery strategies to administer our genomic therapies, including mRNA, AAV, adenovirus, plasmid, and lipid nanoparticles. As more genes and DNA sequences are linked to specific diseases, we believe that the clinical breadth and scope of our ZFP applications will continue to expand.