GENE THERAPY MIGHT BE THE BEST, AND PERHAPS ONLY, CHANCE AT CURING BRAIN DISEASES

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LAWRENCE LAWRY/SCIENCE SOURCE
Dominic Gessler lifts two squirming mice from their cages. Sherri Epstein flinches, but Gessler coaxes her to hold out her hands. He places the small animals in her palms, and they skitter in tiny circles, tickling her skin. “Now,” says Gessler. “Which one used to have Canavan disease?”A few miles away, at Epstein’s home in Worcester, Massachusetts, a young woman lies in bed, her thin limbs bent and still, her curly red hair splayed across a pillow. This is Rachel, Epstein’s 17-year-old daughter, born with Canavan disease—a debilitating, fatal brain disorder. As a newborn, Rachel opened her mouth as if to scream but made no sound. As an infant, she never moved in her sleep. When Rachel failed to lift her head by six months of age, an early intervention therapist suspected she had cerebral palsy. Epstein wishes it had been so. Instead, Rachel had inherited a mutated version of a gene called ASPA from each of her parents. Without a healthy copy of that gene, her cells could not produce a protein needed to break down acid in the brain. So the acid built up, then slowly ate away at the insulation protecting Rachel’s neurons, turning her white matter into a sponge pocketed with fluid-filled sacs. As a result, Rachel does not talk; she cannot see or control her limbs or other bodily functions; she has seizures and will likely not live to adulthood.There is no treatment and no cure for Canavan disease. Yet standing in a room at the University of Massachusetts Medical School, Epstein holds a mouse that once had the same symptoms as Rachel—but now runs effortlessly over her outstretched fingers. Weeks before, the mouse was treated with a single intravenous injection of a gene therapy drug developed in the lab of Gessler’s doctoral adviser, microbiologist Guangping Gao. The drug is the fruit of Gao’s 23-year career in gene therapy. Read more here


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