Article
Treatment of an infant with X-linked severe combined immunodeficiency (SCID-X1) by gene therapy in Australia.
The Medical journal of Australia - 2 May 2005
Ginn Samantha L, Curtin Julie A, Kramer Belinda, Smyth Christine M, Wong Melanie, Kakakios Alyson, McCowage Geoffrey B, Watson Debbie, Alexander Stephen I, Latham Margot, Cunningham Sharon C, Zheng Maolin, Hobson Linda, Rowe Peter B, Fischer Alain, Cavazzana-Calvo Marina, Hacein-Bey-Abina Salima, Alexander Ian E
Abstract excerpt
OBJECTIVE: To report the outcome of gene therapy in an infant with X-linked severe combined immunodeficiency (SCID-X1), which typically causes a lack of T and natural killer (NK) cells. DESIGN AND SETTING: Ex-vivo culture and gene transfer procedures were performed at The Children's Hospital at Westmead, Sydney, NSW, in March 2002. Follow-up to March 2005 (36 months) is available. PATIENT: A 9-month-old male...
Topics
- Antigens, CD34
- Gene Transfer Techniques
- Genetic Therapy
- Genetic Vectors
- Hematopoietic Stem Cells
- Humans
- Infant
- Killer Cells, Natural
- Lymphocyte Count