Why is simply injecting naked DNA fragments usually considered ineffective for gene therapy?

Answer

Cells require a specialized mechanism to effectively take up the new instructions and integrate them or use them to produce the correct protein.

The main obstacle in achieving successful gene therapy is the delivery challenge—ensuring the corrective DNA reaches its intended destination within the target cell, specifically the nucleus. Naked DNA, when injected alone, generally lacks the necessary machinery or mechanism to actively traverse the cell membrane efficiently enough for therapeutic effect. Cells are inherently designed to maintain strict barriers against foreign material. Therefore, delivery vehicles, or vectors, are necessary because they possess the biological capability, evolved through viral mechanisms, to gain entry into the cell and facilitate the use or integration of the genetic payload.

Why is simply injecting naked DNA fragments usually considered ineffective for gene therapy?

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