Gene therapy is experiencing a renaissance, with many of the recent successes in children. For some conditions, the younger the child, the better the genetic correction, because affected tissues degenerate with time. This is the case for adrenoleukodystrophy (ALD), the “Lorenzo’s Oil” disease that strips the insulation from brain neurons. One goal of the not-for-profit Stop ALD is to team gene therapy with newborn screening, to help boys before they begin to lose abilities.
Should gene therapy be attempted even earlier? Before birth?
Fetal gene therapy is already being done in non-human animals, presumably in preparation for phase 1 clinical trials. Gene therapy is technically more challenging than inserting a shunt to drain a hydrocephalic brain or repairing an open spine, because it entails delivering gene-carrying viruses to affected cells and not anywhere else. It is fetal medicine on a different scale.
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