Gene therapy health colege, Summaries of Life Sciences

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GENE THERAPY

Is a medical field which focuses on the utilization of the therapeutic delivery of nucleic acids into a patient’s cells as a drug to treat diseases. Is an experimental technique that uses genes to treat or prevent diseases. In the future, this technique may allow doctors to treat a disorder by inserting a gene into patient’s cells instead of using drugs or surgery. The first gene approved gene therapy clinical research in the US took place on 14 September 1990, at the National Institutes of Health (NIH), under the direction of William French Anderson.

What is gene therapy all about

 There could be unwanted immune system reactions Current gene therapy methods can sometimes target the wrong cells. Gene therapy could cause a potential tumor  The delivery viruses might recover their ability to create diseases It typically works best in single gene mutations

Disadvantage of gene therapy

 One dose of the therapy provides what is essentially a cure for disease-there was a survival rate of 100% of the 18 children involved in the clinical trials of the treatments . The therapy was priced at 594,000 Euros in 2016, with GSK also providing a ‘ money-back guarantee.

Gene therapy success

 Hereditary blindness –gene therapies being developed to treat several different types of inherited blindness-especially degenerative forms where patients gradually lose the light- sensing cells in there eyes.  Hemophilia – people with hemophilia are missing proteins that help their blood form cloths Blood disease Fat metabolism Cancer Parkinson’s disease

 Somatic gene therapy- transfer of a section of DNA to any cell of the body that doesn’t produce sperm or eggs. Effects of gene therapy will not be passed onto patients children.  Germline gene therapy – transfer of a section of DNA to cells that produce eggs or sperm. Effects of gene therapy will be passed onto the patient’s children and subsequent generations.

Types of gene therapy