Tales of biotechnology advances are unlikely to raise a lump in the throat or bring a tear to the eye on their own. Thats what makes Ricki Lewiss recent talk at Lexington Community Education so clever and engrossing. Lewis wrapped a lecture on human genetics and gene therapy in the genuinely moving stories of children whose lives were altered -- some for good, some for bad -- since the field was born in the early 1990s.
As in her book, The Forever Fix: Gene Therapy and the Boy Who Saved It, Lewis talked about the children impacted by rare diseases and the scientists who labored to get closer to a forever fix, a cure that would repair problems at the genetic level.
Lewis is the author of the science textbook Human Genetics: Concepts and Applications, now in its 10th edition and used worldwide, Discovery: Windows on the Life Sciences, and the novel Stem Cell Symphony, about using rock music to activate stem cells in the brain to cure disease. She holds a Ph.D in genetics from Indiana University, and is currently a genetic counselor and teaches at the Alden March Bioethics Institute of Albany Medical College.
She began the lecture with the story of 8-year-old Corey Haas, who in 2008, only four days after undergoing gene therapy to cure his hereditary blindness, screamed in pain when visible sunlight impacts his eyes for the first time.
Lewis uses Coreys story, and how he came to be one of the early beneficiaries of gene therapy, to tell the story of the birth of that miraculous treatment.
Corey was in the right place and the right time, she said. His blindness was treatable but the treatment was experimental.
Gene therapy involves the placement of a gene that a person cant make in their own body into virus cell which is implanted in the body. In Coreys case, his eyes were unable to use Vitamin A, which is necessary for the eye to take in and process light. A virus delivered DNA wrapped in a protein to correct faulty instructions in the affected body part, Lewis said.
Lewis also discussed the story of Jesse Gelsinger, a teenager whose death during gene therapy trials nearly brought the experimental treatment to a close while it was still in its infancy. While Gelsingers story was a tragic failure, Lewis said that scientists were committed to learning from their errors and moving the field further.
One of the most famous children Lewis discussed was David Vetter the so-called Bubble Boy of the 1970s, depicted in the film The Boy in the Plastic Bubble, starring John Travolta. Vetter died of an immunological disease in 1984 at age 30, but the condition is now treatable through gene therapy.
By treating rare diseases such as Vetters, Lewis explained that researchers will learn what they need to treat more common diseases in the future.
Link:
Lewis speaks on gene therapy at Lexington Community Education event
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