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.

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.

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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.