In utero gene editing as a treatment for heritable metabolic syndromes
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DOI: 10.1038/s41574-018-0121-2
Abstract: CRISPRmediated therapeutic editing of metabolic genes. Nat. Med. 24, 1513–1518 (2018) Some heritable diseases manifest during the fetal stage, causing considerable morbidity or mortality after birth. Editing the genome and correcting these mutations in utero… read more here.
Keywords: treatment; gene editing; ht1; fah mice ... See more keywords