Gene Therapy for Cystic Fibrosis :: Science Diseases Medical Essays

Gene Therapy for cystic Fibrosis modernistic molecular ancestrals has given hopes and heartaches to thousands of people around the world. These people be looking towards factor therapy for an answer to their questions. To some people such as NIH director Harold Varmus the answer is a better understanding of basic factortic research and to others the answer is a cure, a hope, that their lethal distemper pass on someday be cured. This essay touches on the background of gene therapy for Cystic Fibrosis (CF), current social and ethical issues facing gene therapy for CF, and some thoughts on the importance of this controversial subject. Gene therapy is the application of the technique where the defect-causing bad genes be replaced by correct reas onenessd genes. The idea of gene therapy is to treat the disease by correcting the bad DNA (Deoxyribonucleic acid) kind of than the current me thod of providing drugs, or proteins non produced by the spoiled gene. Gene therapy address es the problem first hand by directly working with the genetic information causing the disease. From the book influence Genes, Dr. Darryl Macer says It is like f ixing a hole in the bucket, rather than trying to mop up the leaking water. There are two kinds of gene therapy, corporate kiosk gene therapy and germline gene therapy. Somatic cell gene therapy is where genes can be put into specific cells and areas of the body which are alter by the disease. All cellular DNA in our body is basically the same because it was replicated from the same zygote (fertilized egg). Cel ls differentiate into their respective tissues depending upon which part of the bring genome is used. Germline gene therapy is where the correct good gene is inserted into the germline in place of the defective bad gene, and when reproduction occurs the gene will be passed on to the progeny. Inserting the good gene into the very early embryo sta ges of development allows for both germline and somatic cells to b e corrected. Government has limited the research to only somatic cell gene therapy such as performed in Cystic Fibrosis research. Cystic fibrosis is one of the most common lethal mutations in humans. The autosomal recessive allelomorph is carried by 1/20 Caucasians, 1/400 couples will concur children with the disease, and children will be afflicted. If untreated, 95% of affected ch ildren will die to begin with age five (Bell, 1996).