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Paul T Ranum

Children's Hospital of Philadelphia

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Toxicity after AAV delivery of RNAi expression constructs into nonhuman primate brain.

2021 Nature Medicine
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Immortalized striatal precursor neurons from Huntington's disease patient-derived iPS cells as a platform for target identification and screening for experimental therapeutics.

2021 Human Molecular Genetics
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Regulated control of gene therapies by drug-induced splicing.

2021 Nature
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Regulated control of gene therapies with a drug induced switch

2020 bioRxiv
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