gene therapy

The regulatory agencies for medicines in the United States and Europe have issued statements informing about a possible risk of developing certain types of tumors following CAR-T cell immunotherapy treatment. What do we know so far? What is the real risk? Does the benefit-risk balance still hold? Has anything changed after these alerts? We answer these questions with expert opinions and the data currently available. 

Hurler syndrome is a rare and very serious disease caused by an enzyme deficiency, which results in a wide variety of signs and symptoms. Treatment with bone marrow transplantation helps to alleviate some of them, but has little effect on skeletal disorders. Now, a phase I/II trial has tested an autologous transplant of blood stem cells corrected by gene therapy in eight patients. The results, published in the journal Science Translational Medicine, suggest that the treatment is more effective and could also improve these types of disorders. 

Epigenetic editing is a technique that aims to alter gene expression without the need to modify the DNA sequence, as gene editing techniques do. In this way, Italian researchers have succeeded in silencing the PCSK9 gene in mice, thereby reducing cholesterol levels by half for at least a year. According to the authors, and assuming further evaluation is needed, their platform "could lay the foundations for the development of this type of therapy". The results are published in the journal Nature.