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 ...

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 ...

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 ...

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 ...