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In 2013, researchers showed that replacing defective MECP2 genes in mice restored health, using a technique that could also be used in people. The MECP2 Gene Therapy Consortium is a collaboration between four labs who, together, can make the leap from recovered mouse to recovered child.
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Beneficiaries
Situation
Rett Syndrome strikes at random in early childhood, affecting little girls almost exclusively. Each of these girls will lose the ability to walk, talk and use their hands. Most will have difficulty breathing and eating. All are at increased risk of sudden and unexplained death. Many girls live into adulthood, requiring total, 24-hour-a-day care. There is currently no effective treatment.
Solution
The concept behind gene therapy is simple: deliver a healthy gene to compensate for one that is mutated. The genetic target in Rett is known: MECP2. Another advantage is that Rett is not neurodegenerative – neurons don’t die. And we know that restoring t