Tag: <span>hereditary disease</span>

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New DNA repair-kit successfully fixes hereditary disease in patient-derived cells
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New DNA repair-kit successfully fixes hereditary disease in patient-derived cells

by University of Bristol Image of patient derived podocyte kidney cells repaired with novel baculovirus-vectored approach pioneered by the Berger team. Podocin (coloured in green) is restored to the cell surface as in healthy podocytes. Credit: Dr Francesco Aulicino, University of Bristol Genetic mutations which cause a debilitating hereditary kidney disease affecting children and young...

Gene therapy strategy found effective in mouse model of hereditary disease TSC
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Gene therapy strategy found effective in mouse model of hereditary disease TSC

by  Massachusetts General Hospital Credit: CC0 Public Domain Patients with tuberous sclerosis complex, a genetic disorder characterized by the growth of noncancerous tumors in multiple organs of the body, have limited treatment options. A team led by investigators at Massachusetts General Hospital (MGH) has now shown that gene therapy can effectively treat mice that express one...