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Alex Mas Monteys

Children's Hospital of Philadelphia

18

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PIAS1 modulates striatal transcription, DNA damage repair, and SUMOylation with relevance to Huntington's disease.

2021 Proceedings of the National Academy of Sciences of the United States of America
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Author Correction: Striatal neurons directly converted from Huntington's disease patient fibroblasts recapitulate age-associated disease phenotypes.

2020 Nature Neuroscience
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Regulated control of gene therapies with a drug induced switch

2020 bioRxiv
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Selectivity and Biodistribution of WVE-120101, a Potential Antisense Oligonucleotide Therapy for the Treatment of Huntington’s Disease (S56.001)

2017 Neurology
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CRISPR/Cas9 Editing of the Mutant Huntingtin Allele In Vitro and In Vivo

2017 Molecular Therapy
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PIAS1 Regulates Mutant Huntingtin Accumulation and Huntington’s Disease-Associated Phenotypes In Vivo

2016 Neuron
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Reinstating Aberrant mTORC1 Activity in Huntington’s Disease Mice Improves Disease Phenotypes

2015 Neuron
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Artificial miRNAs Targeting Mutant Huntingtin Show Preferential Silencing In Vitro and In Vivo

2015 Molecular therapy. Nucleic acids
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Single nucleotide seed modification restores in vivo tolerability of a toxic artificial miRNA sequence in the mouse brain

2014 Nucleic Acids Research
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A26 Basic Understanding: The Role Of Abnormal Htt Accumulation

2014 Journal of Neurology, Neurosurgery, and Psychiatry
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