Timing of Gene Therapy Interventions: The Earlier, the Better

Abstract excerpt

The concept of gene replacement therapy is quite simple: If you replace a defective gene in a sufficient number of cells and express it at the right level, it will address the root cause of the disease leading to an effective treatment or cure. This somewhat simplistic view does not take into account the secondary pathologies that arise because of the genetic defect, which can cause potentially irreversible...

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