Wednesday, September 21, 2016

Nature Mag - Gene therapy: A new chapter - Sangamo Mention

http://www.nature.com/nature/journal/v537/n7621_supp/full/537S158a.html
Lots of competition !
Great review of LSD programs, here is the Sangamo mention:

The AAVs' payload can be combined with nuclease enzymes for gene editing. This enables the gene to be spliced into the cell's DNA at a predetermined location, reducing the risk of an off-target insertion. Researchers at Sangamo BioSciences in Richmond, California, use AAVs to deliver zinc-finger nucleases and IDUA to liver cells in the patient's body.
“We only have to edit a small number of liver cells, predicted to be less than 1%,” says Michael Holmes, Sangamo's vice-president of research. “That will give a sufficient amount of enzyme to be therapeutic.” As the liver grows, it replicates the edit, generating enzymes throughout the person's life. Sangamo is planning a phase I/II trial involving up to 12 adults with Hurler syndrome this year, and hopes to begin a trial for Hunter syndrome (MPS II) next year.

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