Angelman Syndrome

AAV9-Cas13 gene therapy for Angelman syndrome

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Post published:May 20, 2025
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Research Objective AAV9-Cas13 gene therapy for Angelman syndrome using a first-in-kind mechanism of action that will safely and permanently restore expression of endogenous UBE3A that is deficient in CNS neurons.…

Evaluation of an ex vivo lentiviral gene therapy for the treatment of Angelman syndrome

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Post published:May 20, 2025
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Expression of Ube3a by the hematopoietic system for the treatment of Angelman syndrome

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Post published:May 20, 2025
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