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Transcriptional reprogramming restores UBE3A brain-wide and rescues behavioral phenotypes in an Angelman Syndrome mouse model

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NIAID Data Ecosystem2026-03-14 收录
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https://www.ncbi.nlm.nih.gov/sra/SRP390606
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The specificity and tolerability of a single injection of AAV-S1K therapy for Angelman Syndrome demonstrate the use of ATFs as a promising translational approach for AS.
创建时间:
2023-01-24
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