Gene Therapy for Human Genetic Diseases Has Not Been Successful

Question

Gene therapy for human genetic diseases has not been successful yet.

Accepted Answer

While there have been some successes in small-scale clinical trials, such as with certain types of inherited blindness and severe combined immunodeficiency, overall gene therapy for human genetic diseases has not been widely successful due to challenges in delivering the therapeutic gene to the right cells and avoiding adverse effects. However, research in gene therapy is ongoing and holds promise for the future.