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Non-viral DNA delivery and TALEN editing correct the sickle cell mutation in hematopoietic stem cells

Sickle cell disease is a blood disorder that originates from a single point mutation in the HBB gene that codes for hemoglobin. Here, Moiani et al. developed an efficient TALEN-mediated HBB correction process that is compatible with gene therapy applications.

Arianna Moiani
Gil Letort
Julien Valton
ResearchOpen Access11 Jun 2024
Nature Communications

Volume: 15, P: 1-21
Allogeneic TCRαβ deficient CAR T-cells targeting CD123 in acute myeloid leukemia

CD123, the interleukin-3 receptor alpha chain, is aberrantly expressed in acute myeloid leukemia blasts and leukemia stem cells. Here the authors report the design and characterize the anti-tumor activity of allogeneic CD123-targeted CAR-T cells as a therapeutic approach for acute myeloid leukemia.

Mayumi Sugita
Roman Galetto
Monica L. Guzman
ResearchOpen Access28 Apr 2022
Nature Communications

Volume: 13, P: 1-11
Targeting CD123 in blastic plasmacytoid dendritic cell neoplasm using allogeneic anti-CD123 CAR T cells

Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare and highly aggressive hematologic malignancy derived from the precursors of plasmacytoid dendritic cells. Here the authors characterize the anti-tumor activity of allogeneic anti-CD123 CAR-T cells in preclinical models of BPDCN.

Tianyu Cai
Agnès Gouble
Marina Konopleva
ResearchOpen Access28 Apr 2022
Nature Communications

Volume: 13, P: 1-11
Synchronous and regulated expression of two AU-binding proteins, AUF1 and HuR, throughout murine development

Agnès Gouble
Dominique Morello
Research09 Nov 2000
Oncogene

Volume: 19, P: 5377-5384

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Non-viral DNA delivery and TALEN editing correct the sickle cell mutation in hematopoietic stem cells

Allogeneic TCRαβ deficient CAR T-cells targeting CD123 in acute myeloid leukemia

Targeting CD123 in blastic plasmacytoid dendritic cell neoplasm using allogeneic anti-CD123 CAR T cells

Synchronous and regulated expression of two AU-binding proteins, AUF1 and HuR, throughout murine development

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