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AssigneeUniversity of Washington
InventorNiclas BENGTSSON
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The technology disclosed herein relates to regulatory expression constructs (RC) to drive transgene expression in muscle stem cells (MuSCs). Herein, the MuSC-RC can be used for targeted transgene expression and/or gene editing specifically in MuSCs, which uniquely permits genomic modifications in MuSCs to be inherited by resulting progeny that either differentiate to repair injured muscle, or return to quiescence as MuSCs for future regenerative needs. The technology also relates to nucleic acid constructs comprising the MuSC-RC operatively linked to transgenes, and vectors, compositions and cells comprising the same. Aspects of the technology also relate to methods of expressing a transgene and/or gene editing in a muscle satellite cell using the MuSC-RC, as well as methods for treating a neuromuscular disease or disorder in a subject using the same.
A muscle-stem cell specific regulatory nucleic acid cassette (MuSC-RC) for selectively regulating the expression of an operatively linked target nucleic acid in a muscle stem cell (mSC), the MuSC-RC comprising at least two regulatory elements (REs), wherein the two REs are selected from:
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The nucleic acid construct of claim 2926, wherein expression of the target nucleic acid sequence reduces a pathological effect or symptom of a neuromuscular disease or disorder.
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Dependencies are inferred from the claim text.