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Cure for thalassemia major – from allogeneic hematopoietic stem cell transplantation to gene therapy

Allogeneic hematopoietic stem cell transplantation has been well established for several decades as gene replacement therapy for patients with thalassemia major, and now offers very high rates of cure for patients who have access to this therapy. Outcomes have improved tremendously over the last dec...

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Detalhes bibliográficos
Publicado no:Haematologica
Main Authors: Srivastava, Alok, Shaji, Ramachandran V.
Formato: Artigo
Idioma:Inglês
Publicado em: Ferrata Storti Foundation 2017
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5286930/
https://ncbi.nlm.nih.gov/pubmed/27909215
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3324/haematol.2015.141200
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