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Gene therapy attempts to treat genetic diseases at the molecular level by correcting what is wrong with defective genes. Clinical research into gene therapy’s safety and effectiveness has just begun. No one knows if gene therapy will work, or for what diseases.


Gene therapy was conceptualized in 1972, by authors who urged caution before commencing human gene therapy studies. The first attempt, an unsuccessful one, at gene therapy (as well as the first case of medical transfer of foreign genes into humans not counting organ transplantation) was performed by Martin Cline on 10 July 1980.


The concept of gene therapy arose during the 1960s and 1970s and is still in its infancy, meaning there is a paucity of reliable, long-term data on the safety and efficacy of this therapy. In 1972 ...


It is a gene therapy which has received approval from the European Commission. The Glybera therapy was developed with the intention of treating the problem of familial lipoprotein lipase deficiency (LPLD). When the genes which code for lipoprotein Lipase (LPL) get damaged, this protein doesn't function in a proper manner.


gene therapy trials.{7} However, while FDA has approved gene therapy products for clinical phase investigations in humans as part of an Investigational New Drug (IND) application, the agency has not yet approved a Biological License Application (BLA) allowing for com-mercial distribution of a human gene therapy product.{8,9}


Gene therapy is the addition of new genes to a patient's cells to replace missing or malfunctioning genes. Researchers typically do this using a virus to carry the genetic cargo into cells ...


Gene therapy uses sections of DNA (usually genes) to treat or prevent disease.; The DNA is carefully selected to correct the effect of a mutated gene that is causing disease. The technique was first developed in 1972 but has, so far, had limited success in treating human diseases.


The HGP is like translating an entire library of books written in a foreign language -- the genetic code. But with the "library’s" information, researchers can investigate just what each gene is responsible for in the human body, and how mutations to genes cause genetic diseases.


Gene therapy is designed to introduce genetic material into cells to compensate for abnormal genes or to make a beneficial protein. If a mutated gene causes a necessary protein to be faulty or missing, gene therapy may be able to introduce a normal copy of the gene to restore the function of the protein.


Fixing rare diseases, impressive in its own right, could be just the start. Researchers are studying gene therapy in clinical trials for about 40 to 50 different diseases, says Maria-Grazia ...