Zinc finger nuclease-mediated gene editing in hematopoietic stem cells results in reactivation of fetal hemoglobin in sickle cell disease.

Autor: Lessard, Samuel^1,2 (AUTHOR) samuel.lessard@sanofi.com, Rimmelé, Pauline¹ (AUTHOR), Ling, Hui¹ (AUTHOR), Moran, Kevin¹ (AUTHOR), Vieira, Benjamin¹ (AUTHOR), Lin, Yi-Dong¹ (AUTHOR), Rajani, Gaurav Manohar¹ (AUTHOR), Hong, Vu¹ (AUTHOR), Reik, Andreas³ (AUTHOR), Boismenu, Richard³ (AUTHOR), Hsu, Ben³ (AUTHOR), Chen, Michael³ (AUTHOR), Cockroft, Bettina M.³ (AUTHOR), Uchida, Naoya⁴ (AUTHOR), Tisdale, John⁴ (AUTHOR), Alavi, Asif⁵ (AUTHOR), Krishnamurti, Lakshmanan⁶ (AUTHOR), Abedi, Mehrdad⁷ (AUTHOR), Galeon, Isobelle¹ (AUTHOR), Reiner, David¹ (AUTHOR)

Publikováno v: Scientific Reports. 10/16/2024, Vol. 14 Issue 1, p1-18. 18p.

Aging-like Phenotype and Defective Lineage Specification in SIRT1-Deleted Hematopoietic Stem and Progenitor Cells

Autor: Rimmelé, Pauline ¹, Bigarella, Carolina L. ¹, Liang, Raymond ^{1, 2}, Izac, Brigitte ¹, Dieguez-Gonzalez, Rebeca ¹, Barbet, Gaetan ³, Donovan, Michael ⁴, Brugnara, Carlo ⁵, Blander, Julie M. ^{3, 6}, Sinclair, David A. ⁷, Ghaffari, Saghi ^{1, 2, 6, 8, 9, ∗}

Publikováno v: In Stem Cell Reports 8 July 2014 3(1):44-59

Tento výsledek nelze pro nepřihlášené uživatele zobrazit.
K zobrazení výsledku je třeba se přihlásit.

Ex Vivo Gene-Edited Cell Therapy for Sickle Cell Disease: Disruption of the BCL11A Erythroid Enhancer with Zinc Finger Nucleases Increases Fetal Hemoglobin in Plerixafor Mobilized Human CD34+ Cells

Autor: Moran, Kevin ^{*, 1}, Ling, Hui ^{*, 1}, Lessard, Samuel ^{*, 1}, Vieira, Benjamin ^{*, 1}, Hong, Vu ^{*, 1}, Holmes, Michael C. ^{*, 2}, Reik, Andreas ^{*, 2}, Dang, Denny ^{*, 2}, Gray, David ^{*, 2}, Levasseur, Dana ¹, Rimmele, Pauline ^{*, 1}

Publikováno v: In Blood 29 November 2018 132 Supplement 1:2190-2190

Tento výsledek nelze pro nepřihlášené uživatele zobrazit.
K zobrazení výsledku je třeba se přihlásit.