Adding to the growing body of literature demonstrating the feasibility of correcting lethal genetic diseases before birth, researchers at Children's Hospital of Philadelphia (CHOP) have used DNA base editing in a prenatal ...
Jul 13, 2021
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A rare and controversial mutation in the phospholipase D3 (PLD3) protein—previously linked to Alzheimer's disease—interferes with PLD3's vital recycling function inside neurons. Matthew Schrag of Vanderbilt University ...
Apr 8, 2021
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Cancer starts with mutations in a cell's DNA, but new UC San Francisco research shows that the endurance of a tumor relies on its ability to rapidly evolve and adapt to challenges brought about by the environment in which ...
Apr 2, 2021
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A study aiming to develop a new therapeutic technique could bring a revolution in our approach to treating rare, fatal Sanfilippo syndrome, a disorder that affects children as young as 2 years old and leads to childhood dementia ...
Feb 24, 2021
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A mutation in the CLCN6 gene is associated with a novel, particularly severe neurodegenerative disorder. Scientists from the Leibniz-Forschungsinstitut für Molekulare Pharmakologie (FMP) and the Max Delbrück Center für ...
Nov 19, 2020
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Scientists have revealed the molecular mechanism regulating the trafficking of lysosomes that increases the invasiveness of radioresistant cancer cells following radiotherapy.
Oct 29, 2020
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A versatile probe that can detect with pinpoint accuracy the programmed destruction of defective mitochondria—the powerhouses of cells—has been developed by RIKEN researchers. They used it to show that damaged mitochondria ...
Aug 14, 2020
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Across the globe, approximately 50 million people are living with dementia. The two most common forms are Alzheimer's disease and frontotemporal lobar degeneration (FTLD), which develop when neurons in specific parts of the ...
Jul 9, 2020
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Batten disease is a family of 13 rare, genetically distinct conditions. Collectively, they are the most prevalent cause of neurodegenerative disease in children, affecting 1 in 12,500 live births in the U.S. One of the Batten ...
Jun 30, 2020
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Studying mice, scientists have shown that boosting the activity of specific immune cells in the heart after a heart attack can protect against developing heart failure, an invariably fatal condition. Patients with heart failure ...
Nov 6, 2019
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