Genetherapy.doc

Gene therapy Gene therapy using an? HYPERLINK /wiki/Adenovirus \o Adenovirus adenovirus?vector. A new gene is inserted into a cell using an adenovirus. If the treatment is successful, the new gene will make functional? HYPERLINK /wiki/Protein \o Protein protein?to treat a disease. Gene therapy?is the use of? HYPERLINK /wiki/DNA \o DNA DNA?as a? HYPERLINK /wiki/Biologic_medical_product \o Biologic medical product drug?to treat disease by delivering therapeutic DNA into a patients cells. The most common form of gene therapy involves using DNA that encodes a functional, therapeutic gene to replace a? HYPERLINK /wiki/Mutate \o Mutate mutated?gene. Other forms involve directly correcting a mutation, or using DNA that encodes a therapeutic protein drug (rather than a natural human gene) to provide treatment. In gene therapy, DNA that encodes a therapeutic protein is packaged within a  HYPERLINK /wiki/Vector_(molecular_biology) \o Vector (molecular biology) vector, which is used to get the DNA inside cells within the body. Once inside, the DNA becomes expressed by the cell machinery, resulting in the production of therapeutic protein, which in turn treats the patients disease. Gene therapy was first conceptualized in 1972, with the authors urging caution before commencing gene therapy studies in humans. The first FDA-approved gene therapy experiment in the? HYPERLINK /wiki/United_States \o United States United States?occurred in 1990, when Ashanti DeSilva was treated for HYPERLINK /wiki/Adenosine_deaminase_deficiency \o Adenosine deaminase deficiency ADA- HYPERLINK /wiki/Severe_combined_immunodeficiency \o Severe combined immunodeficiency SCID.?By January 2014, about 2,000 clinical trials had been conducted or had been approved using a number of techniques for gene therapy. HYPERLINK /wiki/Gene_therapy \l cite_note-JGenMed_Database-2  Although early clinical failures led many to dismiss gene therapy as over-hyped, clinical successes si