Gene Therapy Targets Canavan Disease

The Canavan trial signals a new phase in a 10-year offensive that gene therapy researchers have waged against neurodegenerative disorders.

Douglas Steinberg
Sep 16, 2001
Days after learning that their project would survive, thanks to a newly awarded $1.8 million National Institutes of Health grant, researchers at Jefferson Medical College in Philadelphia made medical history on June 5. During a three-hour operation, neurosurgeon Andrew Freese cut six small holes into a girl's skull. Through hair-thin catheters, he then infused areas of her brain with 90 billion virus particles that are expected to infect neurons and express a normal human gene that she lacks. By all accounts, Lindsay Karlin, a 6-year-old afflicted with Canavan disease, thereby became the first person to have recombinant viruses injected into her brain to treat an illness other than cancer.

The Canavan trial signals a new phase in a 10-year offensive that gene therapy researchers have waged against neurodegenerative disorders. Previously limited mostly to cell-culture and animal experiments, the scientists are now poised or starting to take their protocols and...