Gene therapy - promise, pitfalls, and prognosis

Article Abstract:

Gene therapy has not yet been proven effective in treating many diseases, however it is only in its early stages of development. Gene therapy involves using viruses to deliver normal genes to cells with disease-causing genes. In spite of much enthusiasm about gene therapy, two 1995 studies showed that two current gene therapy techniques were not effective in treating Duchenne's muscular dystrophy and cystic fibrosis. However, gene therapy may yet become effective for a number of reasons. It is based on firm scientific fundamentals, and techniques of delivering genes to cells have been improved. For gene therapy to become successful, it must be used on appropriate diseases, deliver genes to several cells, and allow those genes to remain active for a long time. In the 1995 trials, the diseases involve cells throughout the body and the implanted genes were weakened by immune responses. New techniques might involve new delivery viruses and drugs that inhibit immune response.

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Gene therapy for hemophilia

Article Abstract:

Gene therapy that does not use a virus may be an effective treatment for hemophilia. For the first time, measurable amounts of factor VIII were produced in six patients who received the treatment. Unfortunately, factor VIII levels decreased and were undetectable within 10 months.

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Nonviral transfer of the gene encoding coagulation factor VIII in patients with severe hemophila A

Article Abstract:

A type of gene therapy that does not use viruses may be an effective treatment for hemophilia, according to a study of six patients. The gene was introduced into skin cells taken from the patient, which were grown in culture and then injected back into the patient.

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