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Article

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

Blood - 9 Mar 2023

Hardouin Giulia, Antoniou Panagiotis, Martinucci Pierre, Felix Tristan, Manceau Sandra, Joseph Laure, Masson Cécile, Scaramuzza Samantha, Ferrari Giuliana, Cavazzana Marina, Miccio Annarita

Abstract excerpt

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 immunological complications. Therefore, transplantation of autologous, genetically-modified HSPCs is an attractive therapeutic...

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beta-Globins
beta-Thalassemia
CRISPR and Genetic Engineering
CRISPR-Cas Systems
Gene Editing

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Hemoglobinopathies and Related Disorders

Mosquito-borne diseases and control

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Identifiers

PMID: 36508706
DOI: 10.1182/blood.2022016629
PMCID: PMC10651780

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