In rare cases, symptoms of Tay-Sachs and Sandhoff disease appear later in childhood or even in early adulthood, progressing more slowly and offering a longer—but still profoundly challenging ...

Researchers at NIH compared cerebral organoids grown from a Sandhoff disease patient's induced pluripotent stem cells to organoids from cells corrected with CRISPR/Cas9 technology and found a ...

Researchers have identified an FDA-approved drug that could offer new hope for patients with the rare, fatal genetic ...

A conventional strategy for identification of disease genes is to use microarrays ... Use of GINI to detect a known nonsense transcript in Sandhoff cell line 203. The GINI strategy was next ...

Researchers have identified a potential treatment for Sandhoff and Tay-Sachs diseases -- two rare, often fatal lysosomal storage disorders that cause progressive damage to nerve cells in the brain and ...

In a groundbreaking study, researchers at McMaster University have identified a potential treatment for Sandhoff and Tay-Sachs diseases—two rare, often fatal lysosomal storage disorders that cause ...

A team of Canadian researchers has found an existing US Food and Drug Administration-approved drug that could significantly ...