Human gene therapy has been attempted on somatic (body) cells for diseases such as cystic fibrosis, adenosine deaminase deficiency, familial hypercholesterolemia, cancer, and severe combined immunodeficiency (SCID) syndrome.
This is the more common form of gene therapy being done. Germline gene therapy, which involves modifying the genes in egg or sperm cells, which will then pass any genetic changes to future generations. Experimenting with this type of therapy, scientists injected fragments of DNA into fertilized mouse eggs.
Twenty years ago, gene therapy was touted as a cure for everything from cancer to cystic fibrosis. Now it is finally starting to fulfil its promise. In 2012, Glybera became the first gene therapy to be approved, for people with a rare disorder that makes them unable to process dietary fat.
Gene therapy can be used to modify cells inside or outside the body . When it’s done inside the body , a doctor will inject the vector carrying the gene directly into the part of the body that has defective cells.
There are two types of gene therapy treatment: Somatic cell gene therapy and germline therapy . Somatic cell gene therapy involves obtaining blood cells from a person with a genetic disease and then introducing a normal gene into the defective cell (Coutts, 1998).
The methods used for gene therapy include design of therapeutic DNA or RNA constructs, generation of gene transfer vectors, delivery of genes into the target cells, and regulation of transgene expression.
Gene Therapy Types Germline therapy. Somatic gene therapy. Inserting genes into cancer cells . Gene therapy techniques in cancer treatment. Harnessing the immune response. Gene therapy to enhance cancer treatment. Blocking the protection of cancer cells . Pro-drug gene therapy.
Gene therapy offers the possibility of a permanent cure for any of the more than 10,000 human diseases caused by a defect in a single gene . Among these diseases, the hemophilias represent an ideal target, and studies in both animals and humans have provided evidence that a permanent cure for hemophilia is within reach.
Clinical trials of gene therapy in people have shown some success in treating certain diseases, such as: Severe combined immune deficiency. Hemophilia. Blindness caused by retinitis pigmentosa.
The idea of germline gene therapy is controversial . While it could spare future generations in a family from having a particular genetic disorder, it might affect the development of a fetus in unexpected ways or have long-term side effects that are not yet known.
Gene therapy is designed to introduce genetic material into cells to compensate for abnormal genes or to make a beneficial protein. If a mutated gene causes a necessary protein to be faulty or missing, gene therapy may be able to introduce a normal copy of the gene to restore the function of the protein.
Following 18 years of further research, the first gene therapy trial launched in 1990. A four-year-old girl named Ashanthi DeSilva underwent a 12-day treatment for a rare genetic disease known as severe combined immunodeficiency.
Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery.
Current research is evaluating the safety of gene therapy ; future studies will test whether it is an effective treatment option. Several studies have already shown that this approach can have very serious health risks, such as toxicity, inflammation, and cancer.
To date, only 1 gene therapy has been approved in the United States—Luxturna, a treatment for inherited retinal disease that carries a list price of $850,000 —but according to EvaluatePharma, the US healthcare system could see an influx of such therapies in the coming years, with combined sales forecasts of $16 billion