C9ORF72 ZFP-TF

C9ORF72 ZFP-TF is an investigational gene therapy being developed through a collaboration between Sangamo Therapeutics and Pfizer for the treatment of amyotrophic lateral sclerosis (ALS) and frontotemporal lobar degeneration (FTLD) associated with mutations in the C9ORF72 gene. The therapy utilizes engineered zinc finger protein transcription factors (ZFP-TFs) designed to specifically identify and bind to the hexanucleotide repeat expansion (HRE) within the C9ORF72 gene. By fusing a transcriptional repressor domain to the ZFP, the therapy selectively down-regulates the expression of the mutant C9ORF72 allele while preserving the expression of the wild-type allele. This allele-specific silencing is intended to reduce the production of toxic RNA foci and dipeptide repeat proteins (DPRs) that drive neurodegeneration in patients with familial ALS and FTLD.

Other names

C9ORF72 zinc finger protein transcription factorC-9ORF72 zinc finger protein transcription factorC 9ORF72 zinc finger protein transcription factorC9ORF72-ZFPC-9ORF72-ZFPC 9ORF72-ZFP