Gene Therapy Successfully Treats SCID

An infant suffering from X chromosome-linked severe combined immunodeficiency disorder (SCID) that made him too weak to even hold his head up has been restored to a normal life at home following gene therapy.

Before the therapy, the infant was confined to a sterile space and kept alive by a ventilator. A victim of SCID is often called "baby in a bubble” because of the plastic shroud used to preserve sterile conditions around the patient and prevent infection. Standard treatment is a bone marrow transplant but the infant's parents chose the new treatment of gene therapy, in part to obviate the need for chemotherapy, which accompanies bone marrow transplantation.

Doctors took stem cells from the infant's bone marrow and froze them. A bag of the cells was connected to a bag containing a mouse retrovirus genetically engineered to carry a healthy copy of the defective gene in the infant. The virus spliced the good gene into the stem cells and these were reinserted into the infant's bone. Within several months, the infant's lymphocyte count began a rapid rise. Doctors say the infant will need to be followed for many years.

"We're very pleased to announce the first successful cure of a patient in the United Kingdom by gene therapy,” said Professor Christine Kinnon, director of the Center for Gene Therapy of Childhood Disease at the Institute of Child Health (London), who collaborated with doctors at Great Ormond Street Hospital (London) in administering the treatment.




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Institute of Child Health