. Highly efficient endogenous human gene correction using designed zinc-finger nucleases. Nature. 2005 Jun 2;435(7042):646-51. PubMed.

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  1. I think that the general approach is a real tour de force, and could usher in a potential wave of corrective gene therapy, particularly for developmental disorders that can be identified in utero, or for disorders that affect the immune system, where immune cells could be removed, corrected, and reinserted. The relevance to neurodegenerative disease, though, is less clear for several reasons. One problem is that the efficiency of correction is still too low to correct a majority of neurons, although at 7 percent, it is orders of magnitude higher than conventional repair methods. Another problem for neurodegenerative disease is delivery. Even if the efficiency of correction were improved, the efficacy of viral delivery remains low and spatially restricted. Thus, the method faces many challenging hurdles before it could be applied to human neurodegenerative disease.

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  1. A Lucky Break for Gene Therapy: Designer Nuclease Boosts Repair