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A Small Key for a Heavy Door: Genetic Therapies for the Treatment of Hemoglobinopathies

Throughout the past decades, the search for a treatment for severe hemoglobinopathies has gained increased interest within the scientific community. The discovery that ɤ-globin expression from intact HBG alleles complements defective HBB alleles underlying β-thalassemia and sickle cell disease, has...

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Detalles Bibliográficos
Autores principales:	Zittersteijn, Hidde A., Harteveld, Cornelis L., Klaver-Flores, Stefanie, Lankester, Arjan C., Hoeben, Rob C., Staal, Frank J. T., Gonçalves, Manuel A. F. V.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Frontiers Media S.A. 2021
Materias:	Genome Editing
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8525365/ https://www.ncbi.nlm.nih.gov/pubmed/34713239 http://dx.doi.org/10.3389/fgeed.2020.617780

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