Recently, gene therapy has had some very difficult technical and regulatory problems. However, the successful delivery of a therapeutic protein following gene transfer is our best hope for the future. National regulatory boards in conjunction with human gene therapy institutes will establish the safest monitoring protocols possible for this pioneering therapy of human disease. Technical Problems Many of the current technical problems with gene therapy are due to the failure to achieve long-term gene expression with early viral vector systems, a critical requirement for correcting many inborn genetic defects. The toxicity of most gene therapy protocols is due generally not to the introduction of the therapeutic gene, but to the adenoviral vector system.
Investigations using the new vector systems show great promise of fewer complications.
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