What is one of the high-stakes safety concerns associated with integrating vectors used in gene therapy?

Answer

The inserted gene landing near or within an existing critical gene, potentially disrupting normal cell function.

Due to the permanent nature of altering the cellular machinery, safety monitoring is extremely rigorous for gene therapies, particularly those employing integrating vectors like some lentiviruses. The critical risk here involves insertional mutagenesis. If the therapeutic DNA happens to integrate into a location within the host cell’s genome that controls essential cell regulatory pathways—such as a tumor suppressor gene or an oncogene—the insertion itself can inadvertently disrupt normal cellular control mechanisms. This disruption carries the serious consequence of potentially leading to uncontrolled cell growth or triggering the development of cancer.

What is one of the high-stakes safety concerns associated with integrating vectors used in gene therapy?

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