Gene therapy corrects rare blood disease

• April 24, 2024

German researchers reported that through gene therapy (or gene therapy) they managed to correct the malfunction of the gene responsible for the Wiscott-Aldrich syndrome , a disease considered to be rare, but devastating for children because it causes hemorrhages prolonged by small bumps or scratches. This disorder leaves children vulnerable to certain types of cancer and dangerous infections, the news portal publishes. Health Day News.

The experiment was considered successful, because the implanted cells were able to increase the number of platelets and improve some cells of the immune system.

However, one of the 10 children who participated in the study developed an acute T cell leukemia , as a result of the insertion of a healthy gene by means of a viral vector (a modified virus that functions as a vehicle to introduce genetic material into the nucleus of another cell).

The study shows a potential advance in the gene therapy with stem cells in a genetic disorder, said the specialist and director of the Center of Excellence for Aging and Brain Repair at the University of South Florida, Paul Sanberg for Health Day News, although he recognized that it is necessary to find more efficient vectors Y insurance

Children who develop Wiskott-Aldrich syndrome are born with a genetic defect inherited in the X chromosome , which affects the number and size of platelets and makes them more susceptible to bleed easily and suffer infections

The most used treatment for this syndrome is the spinal cord transplant , that of working, can cure the patient, although a dangerous procedure due to the rejection that the body can generate and to the infections that appear later.

Video Medicine: WILL GENE THERAPY CURE US - genetic diseases documentary (April 2024).