(Medical Xpress)— Scientists have discovered that breaking a biological signaling system in an embryo allows them to change the destiny of a cell. The findings could lead to new ways of making replacement organs.
Nov 1, 2012
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New research on two promising gene therapies suggests that combining them into one treatment not only repairs muscle damage caused by Duchenne muscular dystrophy, but also prevents future injury from the muscle-wasting disease. ...
Aug 9, 2013
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The hormone estrogen helps protect memory and promote a healthy brain, but this effect wanes as women age, and even estrogen replacement therapy stops working in humans after age 65. Now researchers at University of Florida ...
Dec 8, 2015
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Diseases caused by genetic mutations in the mitochondria – the powerhouses of the cell – can be disabling, or even deadly. That is why mitochondrial replacement therapy (MRT), otherwise also known as three-person IVF ...
Feb 2, 2015
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Preclinical studies show that gene therapy can improve muscle strength in small- and large-animal models of a fatal congenital pediatric disease known as X-linked myotubular myopathy. The results, appearing in the Jan. 22 ...
Jan 22, 2014
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Treating the rare disease MPS I is a challenge. MPS I, caused by the deficiency of a key enzyme called IDUA, eventually leads to the abnormal accumulation of certain molecules and cell death.
Aug 26, 2014
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In May 2019, the U.S. Food and Drug Administration (FDA) approved a gene replacement therapy for the inherited, progressive neuromuscular disease 5q-linked spinal muscular atrophy (SMA). Approval included all children with ...
Aug 25, 2020
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Four women have successfully had a combined surgery, in which they gave birth by cesarean section and at the same time had surgery to reduce their risk of ovarian cancer, thanks to a team of researchers and clinicians at ...
Nov 3, 2023
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(Medical Xpress)—Gene therapy developed at St. Jude Children's Research Hospital, University College London (UCL) and the Royal Free Hospital has transformed life for men with a severe form of hemophilia B by providing ...
Nov 20, 2014
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Diseases of the eye that cause vision loss and blindness, especially neurodegenerative disorders affecting the retina, are ideal targets for gene therapy, including gene replacement and promising corrective gene editing strategies. ...
Jun 22, 2016
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