Strange disease: She was born without an immune system

An English girl born without an immune system, is being tested for the first gene therapy in the world to "restart" the protective mechanisms in her body and give her the opportunity be able to live in a sterile environment.

Baby Nina Warnell, 17 months old, suffers from a combination of severe immunodeficiency syndrome (SCID) - an inherited disease known as the "bubble child" syndrome , appearing at a rate of 1 / 300,000 young.

Nina has the appearance of a healthy child but her body is unable to resist any kind of bacteria, even the weakest. That means coughing or sneezing can kill you.

Nina was diagnosed in March 2012 after her mother discovered her daughter slept too much and did not eat or drink like other normal children. At 5 weeks of age, Nina was admitted to the hospital and the doctor discovered she had a series of infections, including progressive pneumonia.

Despite the healthy appearance, Nina is very weak because the body does not have an immune system against the invasion of any bacteria.(Photo: healthmedicinet.com)

The incurable disease caused Nina to be cared for in isolation to avoid contact with any other illness. Infections also require her to use a lot of antibiotics and antiviral drugs.

Family members must also disinfect and wear masks, medical gowns before being allowed to enter the air button room to visit Nina. Things get worse when doctors report that she needs an emergency bone marrow transplant to have a chance to survive.

Because both sisters and their parents did not have proper bone marrow, the Warnell family had to call for domestic and foreign charity funds to join hands to find donors.

Over the months, Nina began to recover slowly and eventually gained weight. Doctors also found a way to create an "artificial" immune system for me. Nina must take 15 drugs every day and every 3 weeks, immunoglobulin is required to maintain health, but she is still very weak.

Currently, Professor Bobby Gaspar, a pediatric immunotherapist, has informed the Nina family of the opportunity to treat her with new gene therapy, with a 60 to 90% chance of success. .

Nina lacks a gene that produces an essential enzyme for the birth of a healthy immune system. Therefore, doctors will extract Nina's bone marrow and rebuild it using a new "reprogramming virus" to match her lost key gene into her DNA. This artificial bone marrow will be re-implanted into Nina's body and is expected to help develop a normal immune system.

Nina's father said: "With Nina's condition, you are like facing a timer that counts down every second. If we don't do something within a year, the chances of recovery and survival are very small. It is rare for children to get sick like Nina to survive over 2 years of age ".

Despite knowing that there is a lot of risk in the treatment of genes, Nina's family doesn't have many options and they want to try risking their lives.