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Cystic fibrosis and Portfolio Diseases

Introduction to

Gene Therapy

Many diseases such as cystic fibrosis, muscular dystrophy, haemophilia and cancer are caused by faulty genes. Gene therapy involves the addition of a healthy, working copy of the gene into appropriate cells in the body to replace or override the faulty copy present in the genome.

Why use Gene Therapy to Treat

Cystic Fibrosis?

The basic concept of gene therapy involves introducing a gene into target cells to cure, prevent or slow down the progression of a disease.

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