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 … |
Vox |
Doctors just used a brand new gene therapy to try to save a child’s vision
Vox Now 13, Jack was born with a rare genetic disorder that has eaten away at his vision. At night, he’s fully blind, so before dark he has to leave his friends behind and return to his family’s home in Fair Haven, New Jersey. Jack’s parents had been told … Highly anticipated Luxturna gene therapy for blindness costs $850000Genetic Literacy Project |