Curative gene therapies for rare diseases

Diseases caused by alterations in the DNA can be overcome by providing the cells or tissues with a functional copy of the mutated gene. The most common form of gene therapy implies adding an extra genetic unit into the cell. However, new genome engineering techniques also allow the modification or correction of the existing allele, providing new possibilities, especially for dominant diseases. Gene therapies have been tested for 30 years in thousands of clinical trials, but presently, we have only three authorised gene therapy products for the treatment of inherited diseases in European Union. Here, we describe the gene therapy alternatives already on the market in the European Union and expand the scope to some clinical trials. Additionally, we discuss the ethical and regulatory issues raised by the development of these new kinds of therapies.

Automated summary

Diseases caused by DNA alterations can be treated with gene therapy by providing functional copies of the mutated gene. While gene therapy has been tested for 30 years in thousands of clinical trials, there are currently only three authorized gene therapy products in the European Union for treating inherited diseases.