Sandhoff Study Shows Developmental Dysregulation (IMAGE)

American Society for Biochemistry and Molecular Biology

Sandhoff Study Shows Developmental Dysregulation

Caption

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 surprising overgrowth phenotype early in development. Treatment with a gene therapy vector in early clinical studies for related diseases restored Sandhoff organoids to a normal phenotype.

Credit

Richard Proia, NIDDK

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