Gene Therapy :: Genetics Science Papers

ingredient Therapy A new generation of molecular musicIn September of 1990, eight year old Ashanthi DeSilva make checkup history when she received the first authorized human gene therapy intervention. Ashanthi has been natural with a defective gene that normally produces an essential enzyme adenosine deaminase (adenosine deaminase). If left untreated the inability to produce this enzyme outcomes in the fatal malfunction of the immune system. iv years after receiving the first of many treatment of transgenic cells containing functional ADA genes, Ashanthi was still doing well. Since that first treatment, the National Institutes of Health (NIH) has been spending hundreds of millions of dollars to develop and estimate new gene therapy protocols for a variety of genetic diseases (Marshall 1995). Gene therapy has made a profound impact not only in the treatment of genetic disease moreover also in the way that we pick up at human disease as a whole. The following textbook descri bes the technical and ethical aspects of gene therapy. In the final section a synthesis of the authors personal opinion is presented. 1 Technical AspectsRecombinant DNA (rDNA) engineering has made the transfer of genes from one organism to another possible. The determination of rDNA applied science in conjunction with humans and other mammals is referred to as gene therapy. Gene therapy can be classified into two categories, germinal and somatic (Sections 1.1 & 1.2, respectively). In general, gene therapy involves augmenting the functions of an absent or dysfunctional gene by the demonstration of a functional gene into the cells of the individual. If the transfer is successful, the result is a transgenic individual, who is instanter free of the effects of the dysfunctional gene as long as the new gene is expressed (Griffiths et al. 1996). Gene therapy has been successful in mammal models, and potential applications for treatment of human genetic disease such as Cystic Fibrosis, ADA and various types of cancer are now world aggressively persued by researchers. Clinical trials have already begun (Section 1.4) (Kolberg 1994, Gibbs 1996 & Marshall 1995). 1.1 germinal gene therapy creative gene therapy involves the introduction of genes into both somatic cells and the germline of an individual. The result is not only that the individual is cured of the genetic disease but some of the gametes of the individual may also carry the altered trait. Germinal gene therapy in mice has been successful, but thus far protocols developed for use in mice have not been effective in humans.