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Genome Editing for β-Hemoglobinopathies: Advances and Challenges

β-hemoglobinopathies are the most common genetic disorders worldwide and are caused by mutations affecting the production or the structure of adult hemoglobin. Patients affected by these diseases suffer from anemia, impaired oxygen delivery to tissues, and multi-organ damage. In the absence of a com...

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Detalhes bibliográficos
Publicado no:J Clin Med
Main Authors: Frati, Giacomo, Miccio, Annarita
Formato: Artigo
Idioma:Inglês
Publicado em: MDPI 2021
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7865242/
https://ncbi.nlm.nih.gov/pubmed/33525591
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/jcm10030482
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