Gene therapy is a promising field of medicine that has the potential to treat a wide range of genetic diseases. Over the past few years, there have been several breakthroughs in the development of gene therapies. While most gene therapies are still in the experimental stages, there are a few that have been approved for use. In this article, we will explore gene therapies that are currently available.
Luxturna
Luxturna is a gene therapy that was approved by the FDA in 2017 for the treatment of an inherited retinal disease called Leber congenital amaurosis. The therapy works by delivering a functional copy of the RPE65 gene to the retina, which is responsible for producing a protein necessary for vision. Patients who receive Luxturna have reported improved vision and quality of life.
Zolgensma
Zolgensma is a gene therapy that was approved by the FDA in 2019 for the treatment of spinal muscular atrophy (SMA), a genetic disease that affects muscle strength and movement. The therapy works by delivering a functional copy of the SMN1 gene, which is responsible for producing a protein necessary for motor neuron function. Clinical trials have shown that Zolgensma can improve motor function and increase survival in infants with SMA.
Strimvelis
Strimvelis is a gene therapy that was approved by the European Medicines Agency in 2016 for the treatment of severe combined immunodeficiency (SCID), also known as “bubble boy” disease. The therapy works by modifying the patient’s own cells to produce a functional copy of the ADA gene, which is responsible for producing an enzyme necessary for immune system function. Clinical trials have shown that Strimvelis can improve immune function and increase survival in patients with SCID.
Glybera
Glybera is a gene therapy that was approved by the European Medicines Agency in 2012 for the treatment of a rare genetic disorder called lipoprotein lipase deficiency (LPLD). The therapy works by delivering a functional copy of the LPL gene to the patient’s muscle cells, which is responsible for producing an enzyme necessary for breaking down fats in the blood. Clinical trials have shown that Glybera can reduce the risk of pancreatitis in patients with LPLD.
Conclusion
While gene therapy is still a relatively new field of medicine, there are a few therapies that have been approved for use. Luxturna, Zolgensma, Strimvelis, and Glybera are examples of gene therapies that have shown promising results in clinical trials. As research continues, we may see more gene therapies become available for the treatment of genetic diseases. However, it is important to note that gene therapies are not yet a cure-all and further research is needed to fully understand their potential benefits and risks.