Gene therapy for tuberous sclerosis complex type 2 in a mouse model by delivery of AAV9 encoding a condensed form of tuberin.
Gene therapy for tuberous sclerosis complex type 2 in a mouse model by delivery of AAV9 encoding a condensed form of tuberin. Sci Adv. 2021 Jan; 7(2).
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Anat Stemmer-Rachamimov, M.D.
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Roderick Terry Bronson, D.V.M.
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Bakhos A. Tannous, Ph.D.
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Elizabeth Anne Thiele, Ph.D., M.D.
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Casey A. Maguire, Ph.D.
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Pike-See Cheah, Ph.D.
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Xandra Owens Breakefield, Ph.D.
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Masao Kaneki, Ph.D., M.D.
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Vijaya Ramesh, Ph.D.
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David Joseph Kwiatkowski, M.D., Ph.D.