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Article

In vivo hematopoietic stem cell gene therapy ameliorates murine thalassemia intermedia

13 Nov 2018

Abstract excerpt

Current thalassemia gene therapy protocols require the collection of hematopoietic stem/progenitor cells (HSPCs), in vitro culture, lentivirus vector transduction, and retransplantation into myeloablated patients. Because of cost and technical complexity, it is unlikely that such protocols will be applicable in developing countries, where the greatest demand for a β-thalassemia therapy lies. We have developed a...

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PMID: 30422819
DOI: 10.1172/jci122836

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