In this HemaSphere review, researchers share details about recent progress related to gene therapy technologies and procedures for primary immunodeficiency. Transduction of hematopoietic stem cells using lentiviral vectors has been used to create an effective therapeutic product that can reconstitute many different immune dysfunctions following re-infusion. The authors discuss preclinical studies for challenging targets and emerging technologies that are especially relevant for conditions dependent upon gene regulation for efficacy.

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