Optimized mini-dystrophin genes and expression cassettes and their use
AssigneesThe University of North Carolina at Chapel Hill, Bamboo Therapeutics, Inc.
InventorsXiao Xiao, Juan Li, Chunping Qiao, Scott W. J. McPhee, Richard J. Samulski, Maritza McIntyre
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Abstract
This invention relates to polynucleotides encoding mini-dystrophin proteins, viral vectors comprising the same, and methods of using the same for delivery of mini-dystrophin to a cell or a subject.
Claims15
1-93. (canceled)
A recombinant AAV (rAAV) particle comprising an AAV9 capsid and a vector genome, said genome comprising a first AAV2 inverted terminal repeat (ITR), a muscle-specific transcriptional regulatory element operably linked to a nucleotide sequence encoding a human mini-dystrophin protein consisting of the amino acid sequence of SEQ ID NO:7, a transcription termination sequence, and a second AAV2 ITR.
Dependencies are inferred from the claim text.