Adenine base editor–mediated correction of the common and severe IVS1-110 (G>A) β-thalassemia mutation

β-Thalassemia (BT) is one of the most common genetic diseases worldwide and is caused by mutations affecting β-globin production. The only curative treatment is allogenic hematopoietic stem/progenitor cells (HSPCs) transplantation, an approach limited by compatible donor availability and immunologic...

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Detalles Bibliográficos
Autores principales: Hardouin, Giulia, Antoniou, Panagiotis, Martinucci, Pierre, Felix, Tristan, Manceau, Sandra, Joseph, Laure, Masson, Cécile, Scaramuzza, Samantha, Ferrari, Giuliana, Cavazzana, Marina, Miccio, Annarita
Formato: Online Artículo Texto
Lenguaje:English
Publicado: The American Society of Hematology 2023
Materias:
Gene Therapy
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10651780/
https://www.ncbi.nlm.nih.gov/pubmed/36508706
http://dx.doi.org/10.1182/blood.2022016629

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10651780/
https://www.ncbi.nlm.nih.gov/pubmed/36508706
http://dx.doi.org/10.1182/blood.2022016629

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