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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 111 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
Aime K Johnson,Victoria J McCurdy,Heather L Gray-Edwards,Anne S Maguire, J Nicholas Cochran,Amanda L Gross, Haleigh E Skinner,Ashley N Randle,Jamie L Shirley,Brandon L Brunson,Allison M Bradbury, Stanley G Leroy,
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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Human Gene Therapy (2022)
Terence R. Flotte, Oguz Cataltepe, Ajit Puri,Ana Rita Batista,Richard Moser, Diane McKenna-Yasek, Catherine Douthwright,Gwladys Gernoux,Meghan Blackwood,Christian Mueller, Phillip W. L. Tai,Xuntian Jiang,
Michaël Hocquemiller, Laura Giersch,Xin Mei,Amanda L. Gross,Ashley N. Randle,Heather L. Gray-Edwards,Judith A. Hudson, Sophia Todeasa, Lorelei Stoica,Douglas R. Martin,Miguel Sena-Esteves, Karen Aiach,
Molecular Therapy - Methods & Clinical Development (2022)
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