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The road toward human gene therapy--a 25-year perspective
1Center for Molecular Genetics and Department of Pediatrics, La Jolla, CA 92093-0634, USA. tfriedmann@ucsd.edu
Annals of Medicine
|April 30, 1998
Summary
Human gene therapy, conceptualized in the 1970s, shows significant progress despite limited clinical successes. Advances in gene delivery and expression are driving the field toward its implementation phase.
Area of Science:
- * Medical Science
- * Molecular Biology
- * Genetics
Background:
- * The concept of human gene therapy emerged in the early 1970s.
- * Numerous clinical trials have been conducted in human patients.
- * Gene therapy is a significant driving force in modern medicine.
Observation:
- * Despite extensive research, truly convincing therapeutic successes have been limited.
- * Disappointment stems from exaggerated expectations rather than conceptual failure.
- * Current tools are inadequate for efficient gene delivery, stable expression, and disease correction.
Findings:
- * The fundamental concept of human gene therapy has been a remarkable success.
- * Progress in the field is rapid and continuous.
- * The implementation phase of human gene therapy is anticipated to succeed soon.
Implications:
- * Human gene therapy holds immense potential for treating genetic diseases.
- * Continued advancements in technology will overcome current limitations.
- * The successful implementation phase promises new therapeutic avenues.