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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Fragile X syndrome is a genetic disorder caused by a mutation in a gene that lies at the tip of the X chromosome. It is linked to autism spectrum disorders. People with fragile X experience a range of symptoms that include ...
Jul 27, 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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Researchers from the Department of Molecular Biology and Genetics at Koc University in Turkey and the Pole of Endocrinology, Diabetology and Nutrition, Institute of Experimental and Clinical Research at Université Catholique ...
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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A team of medical researchers affiliated with a host of institutions in the U.S. has used base editing to restore the natural production of the SMN protein in mice, effectively curing spinal muscular atrophy (SMA) in the ...
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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Columbia researchers have discovered how a genetic defect leads to spinal muscular atrophy (SMA), a critical piece of information about the disease that neurologists have been seeking for decades.
Mar 22, 2023
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