Gene Therapy and Genetic Diseases

Abstract

In this paper various methods being used in gene therapy have been described. By means of these methods affected cells are genetically transformed to enable them to develop strength to fight against the diseases. Some of the major methods are cloned human genes and transfer of gene segments, oligonucleotide and synthetic gene, chimeric antibodies, recombined immunized antibodies and vaccines, direct DNA & RNA injection, and transformed antigens. Nowadays various recombined pharmaceutical products are also being developed (and used) by expression cloning. The major ones are: blood coagulating agents, erythropoietin, insulin growth hormone, tissue plasminogen, activator interferon and interleucones, granulosite colony stimulating factor (GCSF) etc. Among the various gene therapy methods major two are: <i>in vivo </i>and <i>ex vivo </i>gene therapy. Direct injection, particle bombardment, receptor mediated endocytosis and liposomes have been described in detail under gene therapy principle. Relevant intramural liposome eLa, gene therapy and hydrodynamics based gene therapy, synthetic chromosomes and angiotensin

gene therapy have also been included.