Therapeutic Genome Editing

Abstract

Efforts to cure single gene disorders by replacing the responsible genes using stem cell transplantation and gene transfer have been pursued for decades. These approaches, however, have been confined to serious diseases due in large part to their toxicity. Somatic gene editing (SGE), which repairs undesirable variants, is changing the landscape. It may well have fewer side effects than prior technologies. Thus, it is possible to consider using this approach to address a much broader array of a single gene variants, including treating milder genetic disorders and even improving function in otherwise healthy people. With these possibilities in mind, we discuss three implications of SGE. The first is the complexity of distinguishing between therapy and enhancement as well as the multifaceted debate about the acceptability of the latter, noting that many in the public are opposed to what they see as unfair advantage. The second, which previously has received little attention, is the tremendous price that is likely to be charged for SGE, which makes the debate about enhancement almost moot because even the needs of the most seriously ill will almost surely not be met, raising serious concerns about equity. The last is ensuring adequate regulation and governance of somatic gene editing.