Gene therapy cures the anemia and lethal bone marrow failure in a mouse model of RPS19-deficient Diamond-Blackfan anemia

Diamond-Blackfan anemia is a congenital erythroid hypoplasia caused by functional haploinsufficiency of genes encoding ribosomal proteins. Mutations involving the ribosomal protein S19 gene are detected in 25% of patients. Enforced expression of ribosomal protein S19 improves the overall proliferati...

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Publicado en:Haematologica
Autores principales: Jaako, Pekka, Debnath, Shubhranshu, Olsson, Karin, Modlich, Ute, Rothe, Michael, Schambach, Axel, Flygare, Johan, Karlsson, Stefan
Formato: Artigo
Lenguaje:Inglês
Publicado: Ferrata Storti Foundation 2014
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Articles
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC4258762/
https://ncbi.nlm.nih.gov/pubmed/25216681
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3324/haematol.2014.111195
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