Somatic gene therapy for genetic disease

Article Abstract:

The identification and cloning of the various genes that are responsible for the known 3,500 human genetic diseases should lead to the development of methods of treatment and management, including prenatal diagnosis, use of gene products, and correction of defective genes with somatic gene therapy. In somatic gene therapy, the defective or absent gene is replaced by its cloned, functioning equivalent. The gene is introduced only into the somatic, or nonreproductive, cells and not the ova or sperm. Hence, the gene cannot be passed to subsequent generations of children. The defective gene may still be present, and the introduction of a functional gene may improve the defective gene, or may cause adverse effects, such as ''silencing'' of normal genes, or activation of other defective genes. However, somatic gene therapy is the most promising method of treating genetic diseases, since complete removal and replacement of a defective gene is a technically difficult process. Cloned genes are introduced into somatic cells by physical and virus-associated methods. The criteria for using somatic gene therapy to treat a genetic disease include: (1) the presence of severe phenotype (physical manifestation or symptoms) associated with the disorder; (2) identification and cloning of the defective gene; (3) decreased need for precise regulation or high levels of expression of the gene product; and (4) a method to implant the genetically modified cells. The genetic diseases involving the hematopoietic or blood-forming cells meet these criteria. Modified genes may be introduced into bone marrow cells using viruses, and the genetically modified cells can be reintroduced by bone marrow transplantation. Ethical aspects such as destruction of bone marrow cells to make room for the transplanted cells; definition of the gene defect to prevent introducing a product that may activate the immune system or cause cancer; and use of somatic gene therapy for diseases that are fatal in infancy or have a poor expected outcome, must be considered. (Consumer Summary produced by Reliance Medical Information, Inc.)

Health aspects, Care and treatment, Methods, Gene therapy, Recombinant DNA, Genetic disorders

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Occurrence, clinical manifestations, and prognosis of Guillain-Barre syndrome

Article Abstract:

Guillain-Barre syndrome is a condition characterized by inflammation in nerves. The cause is unknown, but it may result from infection with certain types of viruses. It causes symptoms of muscle pain, weakness and paralysis. Patients with this syndrome have an abnormally large amount of protein in their spinal fluid. This syndrome is rare, affecting about 1 out of every 60,000 people. It is fatal in fewer than 10 percent of the cases, and most of these deaths are caused by heart or lung failure. To determine the incidence, severity and outcome of this syndrome in children, all of the reported cases of Guillain-Barre syndrome occurring in children in Finland between 1980 and 1986 were reviewed. During this period 13 girls and 14 boys developed the syndrome, giving an overall incidence of 1 case for every 38,000 children under the age of 15. In most of the cases, the disease began in the lower part of the body. Twenty of the affected children had pain in the back or legs for one to two weeks prior to the development of symptoms of nerve impairment. None of the children died and most made full recoveries over a period of several months. Eight of the children required physical therapy for 3 to 22 months after discharge from the hospital. It is concluded that Guillain-Barre syndrome is a rare disease, can cause severe symptoms, may require long periods of hospitalization and therapy, and has a favorable outcome in most cases. (Consumer Summary produced by Reliance Medical Information, Inc.)

Finland, Prognosis, Guillain-Barre syndrome

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