Gene Therapy Reverses Effects of Autism-Linked Mutation in Brain Organoids - University of California San Diego

UC San Diego scientists use lab-grown human brain tissue to identify neural abnormalities in Pitt-Hopkins Syndrome and show gene therapy tools can rescue neural structure and function.

Microscopy images reveal significant differences in size and structure between brain organoids derived from a patient with Pitt-Hopkins Syndrome (right) and from a control (left). In a study publish… [+5978 chars]

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