Gene therapy targets untreatable cystic fibrosis mutation affecting about 10% of patients

Cystic fibrosis is among the most common, known and studied genetic diseases. It affects over 100,000 people worldwide and reduces life expectancy mainly as it causes lung and respiratory problems. Over the years, scientific research has led to the development of several pharmacological therapies for this disease.

This article was originally published on MedicalXpress.com

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