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The first clinical trial introduced a marker gene to a human tumor lymphocytes using a retrovirus. The first gene therapy trial began at the National Institutes Thus, An Introduction to Gene Therapy and Molecular Medicine is a compre- hensive manual that can be used as an aid through the rapidly moving field of gene Gene therapy involves the introduction of one or more foreign genes into an organism to treat hereditary or acquired genetic defects. In gene therapy, DNA. Gene therapy is a methodology for correcting defective genes responsible for disease development. Two methods are available for inserting genetic material. Introduce a non-faulty copy of a gene to stand in for the altered copy causing disease. Later studies led to advances in gene therapy techniques. A newer 4.1 A SHORT INTRODUCTION TO TRANSFER SYSTEMS . GENE THERAPY IN MONOGENIC DISEASES APART FROM PRIMARY. IMMUNODEFICIENCY . In vivo gene therapy means direct introduction of genetic material into the human body. It can be accomplished by use of non-viral vectors. In addition to gene
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