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Gene Therapy for Chronic Granulomatous Disease: Current Status and Future Perspectives

27 Sept 2014

Abstract excerpt

Several Phase I/II clinical trials aiming at the correction of X-linked CGD by gene transfer into hematopoietic stem cells (HSCs) have demonstrated the therapeutic potential of gene modified autologous HSCs for the treatment of CGD. Resolution of therapy-resistant bacterial and fungal infections in liver, lung and spinal canal of CGD patients were clearly documented in all trials. However, clinical benefits were...

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PMID: 25245086
DOI: 10.2174/1566523214666140918113201

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