UNC Pediatrics Delivers Gene Therapy to Hunter Syndrome Patient – Rare Disease Report
Rare Disease Report |
UNC Pediatrics Delivers Gene Therapy to Hunter Syndrome Patient
Rare Disease Report Using Sangamo's zinc finger nuclease (ZFN)-mediated in vivo genome editing technology, the gene therapy inserts a corrective gene into an exact location in the DNA of liver cells. The drug's intention is to enable the liver to produce a lifelong and … |
