Study suggests new molecular strategy for treating fragile X syndrome
Building on more than two decades of research, a study by MIT neuroscientists at The Picower Institute for Learning and ...
MIT Neuroscientists Discover a Tiny Brain Receptor That Could Transform Fragile X Treatment
MIT neuroscientists have made a breakthrough in treating fragile X syndrome by leveraging a novel neurotransmitter signaling ...
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Promising results from first prenatal therapy for spinal muscular atrophy
Spinal muscular atrophy (SMA) is a progressive neurodegenerative disorder set in motion before birth. Scientists at St. Jude ...
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Model born with rare birth defect uses humor to destigmatize limb differences
Lyric Mariah Heard spoke with "Good Morning America" about her life as a model born with a rare birth defect and using humor ...
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Creating the World’s First CRISPR Medicine, for Sickle Cell Disease
In December 2023, through the development efforts of CRISPR Therapeutics and Vertex Pharmaceuticals, their decades-long ...
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CRISPR Unlocks ‘Sleeping’ Genes—A Potential Cure for Prader-Willi Syndrome?
CRISPR epigenome editing successfully reactivates silenced genes in Prader-Willi Syndrome, highlighting potential for ...