If, for instance, a mutation in a certain gene causes the production of a dysfunctional protein resulting in an inherited disease, gene therapy could be used to deliver a copy of this gene that does not contain the deleterious mutation and thereby produces a functional protein.
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Gene therapy was conceptualized in 1972, by authors who urged caution before commencing human gene therapy studies.
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Gene therapy editing is a potential approach to alter the human genome to treat genetic diseases, viral diseases, and cancer.
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Methods for non-viral gene therapy include the injection of naked DNA, electroporation, the gene gun, sonoporation, magnetofection, the use of oligonucleotides, lipoplexes, dendrimers, and inorganic nanoparticles.
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In vivo gene therapy is seen as simpler, since it does not require the harvesting of mitotic cells.
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Gene therapy's led a normal life only with the regular injections performed every two months.
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In 1992, Claudio Bordignon, working at the Vita-Salute San Raffaele University, performed the first gene therapy procedure using hematopoietic stem cells as vectors to deliver genes intended to correct hereditary diseases.
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New gene therapy approach repaired errors in messenger RNA derived from defective genes.
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Gendicine is a cancer gene therapy that delivers the tumor suppressor gene p53 using an engineered adenovirus.
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The therapy used genetically modified T cells to attack cells that expressed the CD19 protein to fight the disease.
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The Gene therapy was designed to increase the levels of SERCA2, a protein in heart muscles, improving muscle function.
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