Spinal muscular atrophy (SMA) is a devastating pediatric neuromuscular disorder caused by loss-of-function mutations in the SMN1 gene, which prevents the body from producing enough of the survival motor neuron (SMN) protein ...
Dec 7, 2023
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Modulating the activity of a kinase in motor neurons may help mitigate mitochondrial defects and other symptoms of spinal muscular atrophy, offering a new therapeutic avenue for the devastating disease, according to a Northwestern ...
Dec 5, 2023
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Researchers at UC Davis Comprehensive Cancer Center have shown that inhibiting a specific protein using gene therapy can shrink hepatocellular carcinoma (HCC) in mice. Silencing the galectin 1 (Gal1) protein, which is often ...
Nov 30, 2023
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Dear Mayo Clinic: My husband and I want to expand our family and are trying to get pregnant with our first baby. I've read about family planning couples can do before conceiving and learned about carrier screening. We don't ...
Oct 19, 2023
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A small molecule previously shown to enhance strength in injured or old laboratory mice does so by restoring lost connections between nerves and muscle fibers, Stanford Medicine researchers have found.
Oct 12, 2023
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More than a billion people around the globe need assistive technology to go about everyday tasks independently. A new artificial intelligence (AI) wheelchair is taking assistive technology a step further and giving people ...
Jul 17, 2023
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Alternative splicing, a clever way a cell generates many different variations of messenger RNAs—single-stranded RNAs involved in protein synthesis—and proteins from the same stretch of DNA, plays an important role in ...
Jun 20, 2023
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My name is Antonio and I suffer from spinal muscular atrophy (SMA, for friends). My life expectancy was only a few years when I was born, almost half a century ago. And all because I "got" a rare, neurodegenerative disease, ...
Jun 15, 2023
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Diffuse intrinsic pontine glioma (DIPG) is a lethal pediatric brain cancer that often kills within a year of diagnosis. Surgery is almost impossible because of the location of the tumors. Chemotherapy has debilitating side ...
Apr 12, 2023
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Scientists in Cambridge and Berlin have used a form of gene therapy to increase levels of the so-called "cold shock protein" in the brains of mice, protecting them against the potentially devastating impact of prion disease.
Mar 24, 2023
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