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Gene therapy for PIDs: Progress, pitfalls and prospects

6 Apr 2013

Abstract excerpt

Substantial progress has been made in the past decade in treating several primary immunodeficiency disorders (PIDs) with gene therapy. Current approaches are based on ex-vivo transfer of therapeutic transgene via viral vectors to patient-derived autologous hematopoietic stem cells (HSCs) followed by transplantation back to the patient with or without conditioning. The overall outcome from all the clinical trials...

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PMID: 23566838
DOI: 10.1016/j.gene.2013.03.098

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