Emerging Therapies

Gene editing gets a head start

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Science  13 Jul 2018:
Vol. 361, Issue 6398, pp. 142
DOI: 10.1126/science.361.6398.142-b

The development of gene-editing technologies into therapies for human disease is an exciting prospect. A crucial question is whether there are advantages to correcting disease-causing mutations before rather than after birth, and whether this approach is even feasible. In a proof-of-concept study in mice, Ricciardi et al. accomplished successful in utero correction of a hemoglobin gene mutation that causes β-thalassemia, a serious blood disorder. They injected nanoparticles containing gene-editing machinery (triplex-forming peptide nucleic acids and single-stranded donor DNA) intravenously into midgestational mouse fetuses. After birth, the treated mice showed sustained amelioration of anemia and survived longer than untreated mice.

Nat. Commun. 10.1038/s41467-018-04894-2 (2018).

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