Gene Therapy
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Definition
Gene Therapy is defined as the insertion of genes into an individual’s cell and biological tissues to treat or cure a disease by replacing or fixing a faulty gene or mutated gene, or delivering a new gene where it is missing. Although the technology is still in its infancy, it has been used with some success, promising significant progress in the treatment, mitigation and potential cure of many diseases, including genetic diseases and cancer. However, these rapidly developing complex technologies are very different to traditional biopharmaceutical products, and bring specific structural characterization challenges.