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Specific correction of pyruvate kinase deficiency-causing point mutations by CRISPR/Cas9 and single-stranded oligodeoxynucleotides.
Front Genome Ed. 2023 Apr 28;5:1104666. doi: 10.3389/fgeed.2023.1104666. eCollection 2023.
Front Genome Ed. 2023.
PMID: 37188156
Free PMC article.
Clinically relevant gene editing in hematopoietic stem cells for the treatment of pyruvate kinase deficiency.
Fañanas-Baquero S, Quintana-Bustamante O, Dever DP, Alberquilla O, Sanchez-Dominguez R, Camarena J, Ojeda-Perez I, Dessy-Rodriguez M, Turk R, Schubert MS, Lattanzi A, Xu L, Lopez-Lorenzo JL, Bianchi P, Bueren JA, Behlke MA, Porteus M, Segovia JC.
Fañanas-Baquero S, et al. Among authors: dessy rodriguez m.
Mol Ther Methods Clin Dev. 2021 May 14;22:237-248. doi: 10.1016/j.omtm.2021.05.001. eCollection 2021 Sep 10.
Mol Ther Methods Clin Dev. 2021.
PMID: 34485608
Free PMC article.
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Gene Editing for Inherited Red Blood Cell Diseases.
Quintana-Bustamante O, Fañanas-Baquero S, Dessy-Rodriguez M, Ojeda-Pérez I, Segovia JC.
Quintana-Bustamante O, et al. Among authors: dessy rodriguez m.
Front Physiol. 2022 Mar 28;13:848261. doi: 10.3389/fphys.2022.848261. eCollection 2022.
Front Physiol. 2022.
PMID: 35418876
Free PMC article.
Review.
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Natural estrogens enhance the engraftment of human hematopoietic stem and progenitor cells in immunodeficient mice.
Fañanas-Baquero S, Orman I, Becerra Aparicio F, Bermudez de Miguel S, Garcia Merino J, Yañez R, Fernandez Sainz Y, Sánchez R, Dessy-Rodríguez M, Alberquilla O, Alfaro D, Zapata A, Bueren JA, Segovia JC, Quintana-Bustamante O.
Fañanas-Baquero S, et al. Among authors: dessy rodriguez m.
Haematologica. 2021 Jun 1;106(6):1659-1670. doi: 10.3324/haematol.2019.233924.
Haematologica. 2021.
PMID: 32354868
Free PMC article.
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Mutations in the RACGAP1 gene cause autosomal recessive congenital dyserythropoietic anemia type III.
Hernández G, Romero-Cortadellas L, Ferrer-Cortès X, Venturi V, Dessy-Rodriguez M, Olivella M, Husami A, De Soto CP, Morales-Camacho RM, Villegas A, González-Fernández FA, Morado M, Kalfa TA, Quintana-Bustamante O, Pérez-Montero S, Tornador C, Segovia JC, Sánchez M.
Hernández G, et al. Among authors: dessy rodriguez m.
Haematologica. 2023 Feb 1;108(2):581-587. doi: 10.3324/haematol.2022.281277.
Haematologica. 2023.
PMID: 36200420
Free PMC article.
No abstract available.
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