Successful gene therapy of Diamond-Blackfan anemia in a mouse model and human CD34+ cord blood hematopoietic stem cells using a clinically applicable lentiviral vector

Diamond-Blackfan anemia (DBA) is an inherited bone marrow failure disorder in which pure red blood cell aplasia is associated with physical malformations and a predisposition to cancer. Twentyfive percent of patients with DBA have mutations in a gene encoding ribosomal protein S19 (RPS19). Our previ...

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Detalles Bibliográficos
Autores principales: Liu, Yang, Dahl, Maria, Debnath, Shubhranshu, Rothe, Michael, Smith, Emma M., Grahn, Tan Hooi Min, Warsi, Sarah, Chen, Jun, Flygare, Johan, Schambach, Axel, Karlsson, Stefan
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Fondazione Ferrata Storti 2021
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Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8804567/
https://www.ncbi.nlm.nih.gov/pubmed/33440921
http://dx.doi.org/10.3324/haematol.2020.269142

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