Somatic Gene Therapy, Paradigm Shift or Pandora’s Box
Gene therapy may be defined as the introduction of genetic material into the cells of a patient in an effort to help cure the disease either by producing a gene product which is missing or in reduced amounts in the patient due to a genetic mutation in the individual (eg Factor VIII protein for haemophilia) or by introduction of new genetic material which either directly or indirectly will help to combat the disease (eg genetic vaccination). Therapeutic genes are delivered using a carrier (called a vector) which may be a non functional viral vector or by using non viral vector approaches such as liposomes or other carrier molecules. All gene therapy protocols involve the introduction of genetic material into cells that have a finite life span such as blood cells, liver cells etc (termed somatic tissue), thus the introduced gene is not passed on to the next generation. This type of gene therapy is known as somatic gene therapy and is in contrast to the concept of germ line gene therapy (which would involve a gene being introduced into sperm or ova so that the gene could be inherited by the children of the patient). Germ line gene therapy is subject to an international moratorium.
KeywordsCystic Fibrosis Gene Therapy Cystic Fibrosis Transmembrane Conductance Regulator Purine Nucleoside Phosphorylase Cystic Fibrosis Transmembrane Conductance Regulator Gene
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