Tag Archives: The Stem Cell Blog

UNC Pediatrics Delivers Gene Therapy to Hunter Syndrome Patient – Rare Disease Report

Continue reading

2018-03-22 NEWS

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

Continue reading

2018-03-22 NEWS

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

all 4 news articles »