Improving gene therapy's tool kit
Marshall, E.
Science 288(5468): 953
2000
ISSN/ISBN: 0036-8075 PMID: 10841711 Document Number: 524291
A French research team has announced the first clear success in a gene transfer experiment. Marina Cavazzana-Calvo and Alain Fischer of the Necker Hospital for Children in Paris, France, transplanted a healthy gene into the bone marrow of 2 children with a rare, lethal immune disorder, allowing the children to leave a protective bubble for the first time. The researchers used one of the oldest vectors employed in gene transfer--a retrovirus vector based on a mouse virus. The use of adenoviruses, adeno-associated viruses, and lentiviruses as vectors for gene therapy is also discussed.