Our cells rely on microscopic highways and specialized protein vehicles to move everything—from positioning organelles to ...

When Columbia neurologist and scientist Neil Shneider speaks to his ALS patients who volunteer for experimental therapies, he's unwaveringly honest.

Developed by Sarepta Therapeutics, Elevidys is a one-time, intravenous treatment that uses adeno-associated virus (AAV) ...

CRISPR-edited iPSCs uncover early mitochondrial defects shared across ALS mutations, revealing pathways that could guide future treatments.

Using the gene scissors CRISPR and stem cells, researchers have managed to identify a common denominator for different gene mutations that all cause the neurological disease ALS. The research shows ...

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Huntington's disease is a genetic disorder that kills nerve cells in the brain, causing people to lose their cognitive and ...

This study proposes a new mechanism by which Chd1 senses and responds to DNA unwrapping via EDBL engagement to suppress ...

Using the gene scissors CRISPR and stem cells, researchers at Stockholm University and the UK Dementia Research Institute (UK DRI) at King's College London have managed to identify a common ...

In a study published in Nature Communications, researchers at the University of Wisconsin–Madison introduced a deep learning ...

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