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Gene therapy for the treatment of a retinal degeneration disease
Gene therapy for the treatment of a retinal degeneration disease
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机译：基因疗法治疗视网膜变性疾病
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摘要
The present invention relates to a polynucleotide configured for the treatment of a retinal degeneration disease, such as retinitis pigmentosa (RP), a nucleic acid vector comprising said polynucleotide, a pharmaceutical composition comprising said nucleic acid vector, a kit comprising said polynucleotide or said nucleic acid vector, a method of making said nucleic acid vector, and a method for treating a retinal degeneration disease.
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