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{ Tag Archives } Genetic Disease

New Enzyme For Treating Rare Genetic Disorder Fabry Disease Shows Promise In Lab

Using a new modelling strategy, researchers in Japan have designed a new enzyme that shows significant promise compared with current therapeutic proteins used to treat patients with Fabry disease, a rare genetic disorder that affects about 1 in 40,000 people and often results in cardiac and [...]

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Fanconi Anemia: Genetically Corrected Blood Cells Obtained From Patients’ Skin Cells

Collaboration research carried out by the teams of Jordi Surrallés, Universitat Autònoma de Barcelona (UAB); Juan Carlos Izpisúa-Belmonte and Angel Raya, Centre for Regenerative Medicine of Barcelona (CMRB); and Juan Antonio Bueren, Centre for Energetic, Environmental and Technological Research (CIEMAT), has resulted in the generation of blood cells from skin cells of patients with a [...]

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