A New Field of Medicine

Genome editing makes permanent changes to the genetic code of a cell by correcting, disabling or modifying the DNA. Genome editing works by using enzymes called “engineered nucleases” that act like molecular scissors. By cutting DNA at a precise spot in the genome, DNA can be removed, added or replaced for therapeutic effect. The goal of genome editing is to provide a permanent therapeutic solution or cure for genetic diseases.

Using Zinc Finger Nucleases to Edit the Genome

Our zinc finger nuclease (ZFN) technology is based on a naturally occurring class of proteins called zinc finger DNA-binding proteins (ZFPs), which recognize and bind to specific sequences of DNA. We engineer ZFNs for precision, efficiency, and specificity, which we believe are the critical parameters for a therapeutic genome editing technology. The ZFNs make a double stranded break in the DNA in a precise location in the albumin gene, where the transgene is permanently integrated using the cell’s natural repair mechanism.

Advantages of ZFNs for designing genome editing therapeutics

  • Precision: Ability to target any desired nucleotide in the human genome
  • Efficiency: Level of editing at the desired target nucleotide or sequence
  • Specificity: Editing the target nucleotide or sequence without editing anywhere else in the genome

OUR IN VIVO GENOME EDITING APPROACH

A ROBUST PIPELINE OF GENOMIC MEDICINES

OUR PIPELINE

Sangamo presentations + Publications

Our broad collection of publications, authored by our own scientists, collaborators, and others, contains original research articles and review articles that are relevant to our ZFP technology and its many applications.

 

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