Selecting patients with sickle cell disease for gene addition or gene editing‐based therapeutic approaches: Report on behalf of a joint EHA Specialized Working Group and EBMT Hemoglobinopathies Working Party consensus conference

Selecting patients with sickle cell disease for gene addition or gene editing‐based therapeutic approaches: Report on behalf of a joint EHA Specialized Working Group and EBMT Hemoglobinopathies Working Party consensus conference

ABSTRACT Sickle cell disease (SCD) remains associated with reduced life expectancy and poor quality of life despite improvements observed in the last decades mostly related to comprehensive care, use of hydroxycarbamide, screening to identify patients at risk of strokes, and implementation of safe t...

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Main Authors: Lucia de Franceschi, Franco Locatelli, David Rees, Christian Chabannon, Jean‐Hugues Dalle, Stefano Rivella, Achille Iolascon, Stephan Lobitz, Miguel R. Abboud, Josu de la Fuente, Pagona Flevari, Emanuele Angelucci, Mariane de Montalembert
Format: Article
Language:English
Published: Wiley 2025-03-01
Series:HemaSphere
Online Access:https://doi.org/10.1002/hem3.70089
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