According to Bulaklak and Gersbach (2020), advances in gene therapy will control the immune system response since about 50% of patients are excluded from treatment due to pre-existing immunity to the viral capsids. One example is the engineering of modified adeno-associated ...Learn More

Since gene therapy involves making changes to a person´s genome, it raises some ethical issues. For example, how to distinguish between what is right or wrong in gene therapy? How to decide which traits are normal and which ones constitute a disease or disability? Will it be ...Learn More

The main obstacles in the development of gene and therapy are manufacturing, affordability, and regulatory issues. ManufacturingIt can be very challenging to grow, source, and transport the cells. For this reason, many companies are developing solutions to overcome this problem ...Learn More

In May 2019, the FDA approved the drug Zolgensma to treat spinal muscular atrophy in children under two years old. In 2017, the 1st gene therapy was approved in the EUA. The drug is Luxturna, used to treat genetic vision loss in patients with Leber congenital amaurosis or ...Learn More

Gene therapy is a technique that uses genes to treat or prevent diseases. With this technique, it is possible to insert genes into a patient´s cells to treat genetic disorders. Gene therapy can reduce the levels of certain disease-causing proteins or increase the production of ...Learn More