The concept is simple: if a mutated gene is causing a problem, replace or supplement it with a new, accurate copy. In theory, such a strategy could not just treat, but cure countless human genetic diseases. In practice, however, developing safe and effective gene therapies has not been easy. Even when identifying a disorder’s genetic basis is fairly straightforward, finding the appropriate delivery vector to target the diseased tissues in the body, while avoiding unintended consequences, has challenged would-be gene therapists for more than 20 years. But more and more researchers are convinced that the technique is on the brink of becoming a common medical practice.
“It’s an incredibly exciting time for the field,” says researcher and medical oncologist David Kirn, founder, president, and chief medical officer at Jennerex, Inc., a San Francisco-based biotherapeutics company that develops and commercializes oncolytic drugs. In the last year alone, he says, major breakthroughs have been published for the use of gene therapy in patients with hemophilia, solid tumors, and leukemia, not to mention the dozens of trials yielding positive results for gene therapies to treat various types of blindness. “It’s just remarkable,” he says. “These decades of work are suddenly really paying off.”
See on the-scientist.com