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UVA researcher developing innovative gene therapy to help children with Rett syndrome news-medical.net - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from news-medical.net Daily Mail and Mail on Sunday newspapers.
Scientist at UVA School of Medicine developing gene therapy to help with Rett syndrome A scientist at the UVA School of Medicine is developing a gene therapy for Rett syndrome. By Daniel Grimes | March 15, 2021 at 6:22 PM EDT - Updated March 15 at 8:46 PM
CHARLOTTESVILLE, Va. (WVIR) - A scientist at the University of Virginia School of Medicine is developing a gene therapy that could help young girls across the country suffering from Rett syndrome.
Rett syndrome is caused by a mutation on the X chromosome. It affects brain development, which can lead to seizures and breathing problems.
Sanchita Bhatnagar is partnering with a scientist at the Center for Gene Therapy to bring about change for those living with Rett syndrome. They aim to manipulate cell regulators to only express healthy copies of certain proteins called microRNA’s.