Development of gene therapies for the treatment of rare genetic diseases

The development of gene therapies for rare genetic diseases focuses on creating innovative treatments that target the underlying genetic causes of these conditions. Rare diseases, often caused by mutations in a single gene, lack effective treatments due to their complexity and low prevalence. Gene therapy aims to correct or replace the faulty gene, offering a potential cure or long-term management of the disease. Techniques such as CRISPR, viral vectors, or gene editing are employed to deliver healthy genes directly into affected cells.