U.S. Department of Health and Human Services

Genetics of Development and Disease Branch

Richard L. Proia, Ph.D., Chief

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Cerebral organoids

Cerebral organoids grown from a Sandhoff disease patient's induced pluripotent stem cells compared to organoids from cells corrected with CRISPR/Cas9 technology show a surprising overgrowth phenotype early in development. Treatment with a gene therapy vector in early clinical studies restored Sandhoff disease organoids to a normal phenotype (http://www.jlr.org/content/59/3/550.full).

Cerebral organoids grown from a Sandhoff disease patient's induced pluripotent stem cells compared to organoids from cells corrected with CRISPR/Cas9 technology.Enlarge