PATHOLOGY

Detailed explanation-1: -Ashanti DeSilva, born in 1986, was 26-months-old when she was diagnosed with SCID after being afflicted by an almost ceaseless series of infections. She suffered from adenosine deaminase (ADA) deficiency, a rarer and slightly less severe form of SCID compared to David Vetter’s X-linked SCID variant.

Detailed explanation-2: -Ashanthi de Silva was the first human to be treated successfully with gene therapy. At the time, in 1990, she was a 4-year-old living with severe combined immunodeficiency (SCID), which is caused by insufficient levels of the enzyme adenosine deaminase (ADA).

Detailed explanation-3: -In the future, genetic therapies may be used to prevent, treat, or cure certain inherited disorders, such as cystic fibrosis, alpha-1 antitrypsin deficiency, hemophilia, beta thalassemia, and sickle cell disease. They also may be used to treat cancers or infections, including HIV.

Detailed explanation-4: -In 1990, 4-year-old Ashanthi de Silva became the first gene therapy success story. She was born with a severe combined immunodeficiency (SCID) due to lack of the enzyme adenosine deaminase (ADA). Without ADA, her T cells died off, leaving her unable to fight infections.