Functional rescue in an Angelman syndrome model following treatment with lentivector transduced hematopoietic stem cells

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Angelman Syndrome is a uncommon neurodevelopmental dysfunction characterised by impaired communication expertise, ataxia, motor and steadiness deficits, mental disabilities, and seizures. The genetic reason for Angelman syndrome is the neuronal lack of UBE3A expression within the mind. A novel method, described right here, is a stem cell gene remedy which makes use of lentivector transduced…