Autologous Stem-Cell-Based Gene Therapy for Inherited Disorders: State of the Art and Perspectives

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Frank J T Staal1, Alessandro Aiuti2,3, Marina Cavazzana4

  • 1Department of Immunohematology and Blood Transfusion (IHB), Leiden University Medical Center, Leiden, Netherlands.

Frontiers in Pediatrics
|November 19, 2019
PubMed

Keywords:
SCIDclinical trialcurative treatmentgene editinggene therapylysosomal storage disordersickle cell diseasethalassemia

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