ENHANCERS DRIVING EXPRESSION IN MOTOR NEURONS

The technology described herein is directed to a gene regulatory element, e.g., enhancer, vectors comprising the same, adeno-associated vectors comprising the same and cells comprising said vectors. In another aspect, described herein are methods of treating a motor neuron disease or disorder compri...

Ausführliche Beschreibung

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Bibliographische Detailangaben
Hauptverfasser: Hrvatin, Sinisa, Nagy, Mark Aurel, Greenberg, Michael E, Griffith, Eric C
Format: Patent
Sprache:eng
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Beschreibung
Zusammenfassung:The technology described herein is directed to a gene regulatory element, e.g., enhancer, vectors comprising the same, adeno-associated vectors comprising the same and cells comprising said vectors. In another aspect, described herein are methods of treating a motor neuron disease or disorder comprising administration of said vectors, e.g., AAV vectors. In another aspect, described herein are nucleic acid compositions comprising the gene regulatory element as described herein.