ZINC FINGER NUCLEASE VARIANTS FOR TREATING OR PREVENTING LYSOSOMAL STORAGE DISEASES

The present disclosure provides 2-in-1 zinc finger nuclease variants and methods of treating and/or preventing a lysosomal storage disorder using said zinc finger nuclease variants.

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Bibliographische Detailangaben
Hauptverfasser: Huston, Marshall, De Alencastro, Gustavo, Shivak, David
Format: Patent
Sprache:eng
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Beschreibung
Zusammenfassung:The present disclosure provides 2-in-1 zinc finger nuclease variants and methods of treating and/or preventing a lysosomal storage disorder using said zinc finger nuclease variants.