Minor splicing defects of Pcyt2 constitute a major pathogenic factor for SMA. (IMAGE)

Science China Press

Minor splicing defects of Pcyt2 constitute a major pathogenic factor for SMA.

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Pcyt2 is specifically expressed in subsets of neuroglia. Replacing the Pcyt2 minor intron with a major intron in the Drosophila SMA model substantially reverses multiple SMA‑associated pathological phenotypes.

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©Science Bulletin

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