Genetic Syndrome & Gene Therapy

Gene therapy targets the faulty genes responsible for genetic diseases. Inheriting a faulty (mutated) gene can directly cause a wide range of disorders such as cystic fibrosis and hemophilia. It can also cause susceptibility to some cancers. Gene therapy can be used to replace a faulty gene with a healthy version or to introduce a new gene that can cure a condition or modify its effects.

 

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