Potential Genome-editing Therapy Shows Promise in Fabry Mouse Model

Potential Genome-editing Therapy Shows Promise in Fabry Mouse Model

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Potential Genome-editing Therapy Shows Promise in Fabry Mouse Model

An experimental genome-editing therapy safely and effectively increased levels of alpha-galactosidase A (Gal A) — which is lacking in people with Fabry disease — in a mouse model of the disease, a study has shown. By only changing the genome of about 10% of cells in the liver — where Gal A is produced — the approach was able to resolve the toxic globotriaosylceramide (Gb3) accumulation in all key tissues that are damaged in Fabry.…

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