Introduction to Gene Therapy: It Sounds Simple, But It’s Sure Not Easy

Muscular dystrophy, hemophilia, Leber congenital amaurosis (LCA), cancer… they all have one thing in common. They are genetic diseases, ultimately caused by missing or malfunctioning genes in the patient’s DNA. Until recently, we could treat the symptoms of many genetic diseases, but not the causes. There was no way to reach inside a person’s genes and repair the faulty code that started the whole problem. Research into gene therapy aims to change that. What is gene therapy? Genes are blueprints for proteins. A complete set of working proteins makes a working human. In contrast, missing or malfunctioning proteins sometimes lead […]

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