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AVR-RD-04 is an investigational gene therapy for cystinosis. It consists of autologous CD34+ enriched cells transduced with a lentiviral vector containing RNA resulting in codon-optimized cDNA encoding for functional human cystinosin.
AVR-RD-04 is an investigational gene therapy for cystinosis. It consists of autologous CD34+ enriched cells transduced with a lentiviral vector containing RNA resulting in codon-optimized cDNA encoding for functional human cystinosin.
每项发明一条，涵盖美国、欧洲、PCT 与中国的同族专利。
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