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ABO gene editing for the conversion of blood type A to universal type O in Rh donor-derived human-induced pluripotent stem cells
Petazzi, Paolo (Institut Germans Trias i Pujol. Institut de Recerca contra la Leucèmia Josep Carreras)
Miquel-Serra, Laia (Hospital Universitari Vall d'Hebron)
Huertas, Sergio (Hospital Universitari Vall d'Hebron)
González, Cecilia (Hospital Universitari Vall d'Hebron)
Boto, Neus (Laboratorio de Immunohematología (Barcelona))
Muñiz-Diaz, Eduardo (Hospital Universitari Vall d'Hebron)
Menéndez Bujan, Pablo (Institut Germans Trias i Pujol. Institut de Recerca contra la Leucèmia Josep Carreras)
Sevilla, Ana (Universitat de Barcelona)
Nogues Galvez, Nuria (Hospital Universitari Vall d'Hebron)
Universitat Autònoma de Barcelona

Date: 2022
Abstract: The limited availability of red cells with extremely rare blood group phenotypes is one of the global challenges in transfusion medicine that has prompted the search for alternative self-renewable pluripotent cell sources for the in vitro generation of red cells with rare blood group types. One such phenotype is the Rh, which lacks all the Rh antigens on the red cell membrane and represents one of the rarest blood types in the world with only a few active blood donors available worldwide. Rh red cells are critical for the transfusion of immunized patients carrying the same phenotype, besides its utility in the diagnosis of Rh alloimmunization when a high-prevalence Rh specificity is suspected in a patient or a pregnant woman. In both scenarios, the potential use of human-induced pluripotent stem cell (hiPSC)-derived Rh red cells is also dependent on ABO compatibility. Here, we present a CRISPR/Cas9-mediated ABO gene edition strategy for the conversion of blood type A to universal type O, which we have applied to an Rh donor-derived hiPSC line, originally carrying blood group A. This work provides a paradigmatic example of an approach potentially applicable to other hiPSC lines derived from rare blood donors not carrying blood type O.
Rights: Aquest document està subjecte a una llicència d'ús Creative Commons. Es permet la reproducció total o parcial, la distribució, la comunicació pública de l'obra i la creació d'obres derivades, fins i tot amb finalitats comercials, sempre i quan es reconegui l'autoria de l'obra original. Creative Commons
Language: Anglès
Document: Article ; recerca ; Versió publicada
Subject: ABO ; CRISPR ; Gene edition ; Ipsc ; Rare blood groups ; Rh ; Transfusion medicine
Published in: Clinical and Translational Medicine, Vol. 12 (october 2022) , ISSN 2001-1326

DOI: 10.1002/ctm2.1063
PMID: 36281739


15 p, 3.3 MB

The record appears in these collections:
Research literature > UAB research groups literature > Research Centres and Groups (research output) > Health sciences and biosciences > Institut d'Investigació en Ciencies de la Salut Germans Trias i Pujol (IGTP) > Josep Carreras Leukaemia Research Institute
Articles > Research articles
Articles > Published articles

 Record created 2023-07-12, last modified 2026-03-14



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