Genetic Polymorphisms Associated with Fetal Hemoglobin (HbF) Levels and F-Cell Numbers: A Systematic Review of Genome-Wide Association Studies
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Autori:
Coralea Stephanou, Stephan Menzel, Sjaak Philipsen, Petros Kountouris
Pubblicato in:
International Journal of Molecular Sciences, Numero 25, 2025, ISSN 1422-0067
Editore:
MDPI AG
DOI:
10.3390/ijms252111408
A prime editing strategy to rewrite the γ-globin promoters and reactivate fetal hemoglobin for sickle cell disease
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Autori:
Anne Chalumeau, Maria Bou Dames, Letizia Fontana, Simone Amistadi, Panagiotis Antoniou, Priyanka Loganathan, Margaux Mombled, Guillaume Corre, Martin Peterka, Mario Amendola, Carine Giovannangeli, Marcello Maresca, Annarita Miccio, Mégane Brusson
Pubblicato in:
Blood, Numero 146, 2025, ISSN 0006-4971
Editore:
American Society of Hematology
DOI:
10.1182/BLOOD.2024028166
Emerging lipid–polymer hybrid nanoparticles for genome editing
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Autori:
Mariana Gameiro; João F. Mano; Vítor M. Gaspar
Pubblicato in:
Polymer Chemistry, Numero 15, 2024, ISSN 1759-9962
Editore:
The Royal Society of Chemistry
DOI:
10.1039/D4PY00298A
Enhanced γ-globin reactivation and sickle cell correction through a repressor-to-activator motif switch in the HBG1/2 promoters
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Autori:
Anne Chalumeau, Panagiotis Antoniou, Maria Bou Dames, Pierre Martinucci, Elena Retana, Pragya Gupta, Mike Firth, Muralidhar Reddivari, Aathira Sarath Chandran, Jonathan S. Yen, Martin Peterka, Marcello Maresca, Mégane Brusson, Annarita Miccio
Pubblicato in:
Molecular Therapy Nucleic Acids, Numero 37, 2026, ISSN 2162-2531
Editore:
Elsevier BV
DOI:
10.1016/J.OMTN.2026.102974
Reactivation of developmentally silenced globin genes through forced linear recruitment of remote enhancers
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Autori:
Anna-Karina Felder, Sjoerd J. D. Tjalsma, Han J. M. P. Verhagen, Rezin Majied, Marjon J. A. M. Verstegen, Thijs C. J. Verheul, Jeffrey van Haren, Rebecca Mohnani, Richard Gremmen, Peter H. L. Krijger, Sjaak Philipsen, Emile van den Akker, Wouter de Laat
Pubblicato in:
Blood, Numero 146, 2025, ISSN 0006-4971
Editore:
American Society of Hematology
DOI:
10.1182/BLOOD.2024028128