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个人简介
The Sena-Esteves laboratory is investigating gene therapy approaches for the treatment of neurodegenerative lysosomal storage diseases such as GM1-gangliosidosis and GM2-gangliosidoses (Tay-Sachs and Sandhoff diseases). We have devised new ways to deliver therapeutic levels of the missing enzymes to the entire brain by injection of adeno-associated virus (AAV) vectors into specific structures in the CNS. Based on the exceptional results that we have obtained in animal models, we are initiating pre-clinical studies that will culminate in a human clinical trial for Tay-Sachs disease. Similar work is planned for GM1-gangliosidosis.
研究兴趣
论文共 187 篇作者统计合作学者相似作者
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Minggang Fang, Sara K. Deibler,Pranathi Meda Krishnamurthy,Feng Wang,Paola Rodriguez, Shahid Banday, Ching-Man Virbasius,Miguel Sena-Esteves,Jonathan K. Watts,Michael R. Green
FRONTIERS IN NEUROSCIENCE (2024): 1348478-1348478
Alex J. Roy, Jeffrey R. Leipprandt,Joseph R. Patterson,Anna C. Stoll, Christopher J. Kemp, Zaipo-Tcheisian D. Oula, Tyler Mola, Ana R. Batista,Caryl E. Sortwell,Miguel Sena-Esteves, Richard R. Neubig
ASPET 2024 Annual Meeting Abstract - Cellular and Molecular Pharmacology (2024)
Salome Funes,Jonathan Jung,Del Hayden Gadd,Michelle Mosqueda,Jianjun Zhong, Shankaracharya,Matthew Unger,Karly Stallworth, Debra Cameron,Melissa S. Rotunno,Pepper Dawes,Megan Fowler-Magaw,
Nature Communicationsno. 1 (2024): 1-25
Annals of neurology (2023)
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Tyler Mola,Hannah Villeneuve, Dasha Khvorova-Wolfson,Simon Wentworth, Pamela St Louis,Rrita Daci,Ana Rita Batista,Miguel Sena-Esteves
MOLECULAR THERAPYno. 4 (2023): 352-352
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Rrita Daci, Pamela St Louis, Tyler Mola,Hannah Villeneuve, Julie Aguiar, Rob King, Heather L. Gray-Edwards,Ana Rita Batista,Miguel Sena-Esteves
MOLECULAR THERAPYno. 4 (2023): 79-80
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Annals of Neurologyno. 5 (2023): 969-986
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Human Gene Therapy (2022)
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