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Molecular and Cellular Pediatrics|November 16, 2018
Gene correction of HBB mutations in CD34<sup>+</sup> hematopoietic stem cells using Cas9 mRNA and ssODN donorsJustin S Antony, Ngadhnjim Latifi, A K M Ashiqul Haque, et al.
Blood Advances|January 3, 2022
Blinatumomab in pediatric relapsed/refractory B-cell acute lymphoblastic leukemia: RIALTO expanded access study final analysisFranco Locatelli, Gerhard Zugmaier, Noemi Mergen, et al.
British Journal of Haematology|June 25, 2016
Treatment of graft failure with TNI-based reconditioning and haploidentical stem cells in paediatric patientsHeiko-Manuel Teltschik, Frank Heinzelmann, Bernd Gruhn, et al.
Genes|December 23, 2022
Challenges in Gene Therapy for Somatic Reverted Mosaicism in X-Linked Combined Immunodeficiency by CRISPR/Cas9 and Prime EditingYujuan Hou, Guillermo Ureña-Bailén, Tahereh Mohammadian Gol, et al.
British Journal of Haematology|June 29, 2006
Safe adoptive transfer of virus-specific T-cell immunity for the treatment of systemic adenovirus infection after allogeneic stem cell transplantationTobias Feuchtinger, Susanne Matthes-Martin, Celine Richard, et al.
Genes|January 21, 2022
Somatic Reversion of a Novel <i>IL2RG</i> Mutation Resulting in Atypical X-Linked Combined ImmunodeficiencyYujuan Hou, Hans Peter Gratz, Guillermo Ureña-Bailén, et al.
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