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Gene therapy for primary immunodeficiencies

18 Dec 2020

Abstract excerpt

Primary immunodeficiencies (PIDs) are a group of rare inherited disorders of the immune system. Many PIDs are devastating and require a definitive therapy to prevent progressive morbidity and premature mortality. Allogeneic haematopoietic stem cell transplantation (alloHSCT) is curative for many PIDs, and while advances have resulted in improved outcomes, the procedure still carries a risk of mortality and...

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PMID: 33336808
DOI: 10.1111/bjh.17269

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