Preventing Mitochondrial Disease

A Path Forward

Abstract

In a possible first, the heritable transmission of a fatal mitochondrial DNA disease (Leigh syndrome) may have been prevented by replacing the mutation-bearing mitochondria of oocytes with donated mutation-free counterparts. The procedure, carried out by a U.S.-led team, took place in Mexico in circumvention of a statutory U.S. moratorium on mitochondrial replacement. This development calls into question the regulatory utility of a national moratorium in a globalized world wherein cross-border care is increasingly prevalent. This development also calls to account the moral defensibility of a moratorium that acquiesces in the birth of gravely ill children whose afflictions could have been prevented. In this Current Commentary, we outline a potential path forward by analyzing the dual imprint of the moratorium, examining its legislative shortcomings, exploring its motivational roots, considering its national effect, and proposing its unlinking from the related yet distinct ban on editing the genome of the human embryo.

In Brief

The birth of neonates afflicted with preventable mitochondrial diseases proceeds unabated in the United States as a result of an ill-defined statutory moratorium, the redress of which is herein delineated.