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GENE THERAPY VECTORS FOR TREATMENT OF DANON DISEASE
GENE THERAPY VECTORS FOR TREATMENT OF DANON DISEASE
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机译:基因治疗载体治疗Danon病
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摘要
The disclosure relates to optimized polynucleotide sequences for LAMP-2B, expression cassettes, vectors, and methods of use thereof in treating disease, e.g. Danon disease.
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