Gene therapy

Enzymes fix disease genes

    Genome-editing enzymes can be used to correct patient-specific genetic defects.

    Mutations in the COL7A1 gene cause the skin-blistering disease recessive dystrophic epidermolysis bullosa, which can be fatal. A team led by Jakub Tolar at the University of Minnesota in Minneapolis engineered enzymes called transcription activator-like effector nucleases (TALENs) to repair the defective part of the gene in skin cells from a patient with the disease. These corrected cells were then reprogrammed into pluripotent stem cells, which, the authors suggest, could be developed into more-specialized cells that are suitable for therapy.

    Mol. Ther. (2013)

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    Enzymes fix disease genes. Nature 497, 8 (2013).

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