Article
Generation of dyskeratosis congenita-like hematopoietic stem cells through the stable inhibition of DKC1.
Stem cell research & therapy - 29 Jan 2021
Carrascoso-Rubio Carlos, Zittersteijn Hidde A, Pintado-Berninches Laura, Fernández-Varas Beatriz, Lozano M Luz, Manguan-Garcia Cristina, Sastre Leandro, Bueren Juan A, Perona Rosario, Guenechea Guillermo
Abstract excerpt
Dyskeratosis congenita (DC) is a rare telomere biology disorder, which results in different clinical manifestations, including severe bone marrow failure. To date, the only curative treatment for the bone marrow failure in DC patients is allogeneic hematopoietic stem cell transplantation. However, due to the toxicity associated to this treatment, improved therapies are recommended for DC patients. Here, we aimed...
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