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Hematopoietic stem cell gene therapy

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Company Genethon
DescriptionHematopoietic cells genetically modified with a lentiviral vector containing the cytochrome b-245 (p91phox)
Molecular Target
Mechanism of ActionGene therapy
Therapeutic ModalityGene therapy: Viral vector: Lentivirus
Latest Stage of DevelopmentPreclinical
Standard IndicationChronic granulomatous disease (CGD)
Indication DetailsTreat chronic granulomatous disease (CGD)
Regulatory Designation
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