Gene Therapy – A Revolution in Progress: Human Genetics …

Gene therapy attempts to treat genetic diseases at the molecular level by correcting what is wrong with defective genes. Clinical research into gene therapys safety and effectiveness has just begun. No one knows if gene therapy will work, or for what diseases. If gene therapy is successful, it could work by preventing a protein from doing something that causes harm, restoring the normal function of a protein, giving proteins new functions, or enhancing the existing functions of proteins. How Do You Do It? Gene therapy relies on finding a dependable delivery system to carry the correct gene to the affected cells. The gene must be delivered inside the target cells and work properly without causing adverse effects. Delivering genes that will work correctly for the long term is the greatest challenge of gene therapy.

Human ex vivo Gene Therapy

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Gene Therapy - A Revolution in Progress: Human Genetics ...