Q.Who was the first patient who was given gene therapy? Why was the given treatment recurrent in nature?

The first patient to receive gene therapy was a four-year-old girl suffering from Severe Combined Immunodeficiency (SCID) due to a deficiency of the enzyme adenosine deaminase (ADA). This treatment was carried out in 1990, though the NCERT textbook does not name the patient.

The treatment was recurrent because it involved a two-step process that did not provide a permanent cure. First, lymphocytes from the patient's blood were removed and genetically engineered in the lab using a retrovirus to insert a functional ADA gene. These corrected cells were then infused back into the patient. However, since lymphocytes are not self-renewing stem cells, they have a limited lifespan. Therefore, the patient req …

The story of the first gene therapy patient is not just a medical milestone—it is a deeply human one. In September 1990, a four-year-old girl named Ashanti DeSilva became the first person to receive gene therapy as a treatment for a life-threatening genetic disorder. Ashanti suffered from Severe Combined Immunodeficiency (SCID), a condition caused by a deficiency of the enzyme adenosine deaminase (ADA). This enzyme is crucial for the proper functioning of immune cells, particularly T-lymphocytes. Without it, her immune system was virtually non-existent, leaving her vulnerable to every infection. Children with this condition often have to live in sterile "bubbles" to avoid germs, which is why SCID is sometimes called "bubble boy disease."

The treatment involved a groundbreaking approach. Doctors removed some of Ashanti's own blood cells, specifically her T-lymphocytes, and used a disabled retrovirus to insert a functional copy of the ADA gene into these cells. The corrected cells were then multiplied in the lab and infused back into her body. This was the first time a human being had received a therapeutic gene directly into their cells.

Now, why was this treatment recurrent in nature? The answer lies in the type of cells that were corrected. In Ashanti's case, the gene therapy was performed on her T-lymphocytes, which are mature immune cells. These cells have a limited lifespan—they circulate in the blood for weeks or months, then die naturally. They are not stem cells, which can divide and replenish themselves indefinitely. So, while the corrected T-cells could fight infections for a while, they eventually wore out. To maintain a working immune system, Ashanti needed repeated infusions of freshly corrected T-cells every few months. …

Method — separate WHO from WHY-recurrent

Who: a 4-year-old girl treated in 1990 for SCID caused by ADA deficiency (the NCERT textbook doesn't name her, though she is historically known as Ashanti DeSilva). …