Heartbreaking discussion of MPS-3 patient really clarifies the need for SGMO to get their program into the clinic. Also spends most of the article talking CRISPR's.
Sangamo Excerpt:
The easiest sorts of gene therapy will be those that can be done outside the body—ex vivo, in lab speak. The appeal of ex vivo work is the level of control; cells can be extracted, have their genes manipulated, and have their new genes tested before being put back. To see the sort of things that this makes possible take a look at the work being done by Sangamo Biosciences, based in Richmond, California, which has been working for a decade on an earlier, more cumbersome gene-editing technology that makes use of what are known as “zinc fingers”. It is trying to apply that technology to beta-thalassaemia, sickle-cell disease, haemophilia and HIV infection.
In clinical trials of its HIV treatment, Sangamo takes the immune cells that the virus infects out of the patient’s bloodstream and edits in a mutation that makes them highly resistant to infection. It then grows up a large number of the edited cells and infuses them back into the patient, where it is hoped they will flourish. A similar sort of approach can also be used in blood disorders such as beta-thalassaemia and sickle-cell disease which are caused by mutations in the globin gene. The idea is to extract blood stem cells from bone marrow, edit them so as to switch on the production of fetal haemoglobin (which the body stops producing shortly after birth, even if it cannot make the adult stuff) and return the stem cells to the body. It would be like a bone-marrow transplant—except that since the new genetically improved cells come from the patient’s own body there is no danger of rejection.
http://www.economist.com/news/briefing/21661799-it-now-easy-edit-genomes-plants-animals-and-humans-age-red-pen
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