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Preliminary Results Point to Success of In Vivo Gene Editing
Preliminary Results Point to Success of In Vivo Gene Editing
Two studies show signs that the introduced DNA is functioning, but it’s too early to know if patients actually benefit.
Two studies show signs that the introduced DNA is functioning, but it’s too early to know if patients actually benefit.
hunter syndrome
Early Results of First In Vivo Gene-Editing Trial Encouraging
Kerry Grens | Sep 5, 2018 | 2 min read
Two patients who received Sangamo’s zinc finger–based treatment for Hunter syndrome have lower biomarkers of the condition, but no signs of new enzyme production.
First In Vivo Human Genome Editing to Be Tested in New Clinical Trial
Abby Olena, PhD | May 18, 2017 | 4 min read
Sangamo Therapeutics will use zinc finger nucleases to introduce the gene for a missing clotting factor into the livers of men with hemophilia B.