New virus offers gene therapy a boost . . . . . . . . . . . . . . .
The goal of gene therapy is to treat disease by delivering a healthy gene to people whose copy has been lost or damaged, rather than by prescribing a drug. The advantage is that the gene becomes part of the chromosome and produces the missing protein. The challenge for gene therapy is delivering enough of the healthy gene to exert a therapeutic effect. If the new results with AAV1 in mice translate to humans, there would be 20 times more Factor IX than a person needs, says Richard Samulski, of the University of North Carolina at Chapel Hill. To the researchers' surprise, AAV1 and AAV5 were able to infect and deliver genes to slow and to fast twitch muscle types.