Generation of dyskeratosis congenita-like hematopoietic stem cells through the stable inhibition of DKC1.

ABSTRACT: 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 at generating DC-like human hematopoietic stem cells in which the efficacy of innovative therapies could be investigated. Because X-linked DC is the most frequent form of the disease and is associated with an impaired expression of DKC1, we have generated DC-like hematopoietic stem cells based on the stable knock-down of DKC1 in human CD34⁺ cells with lentiviral vectors

SUBMITTER: Carrascoso-Rubio C 

PROVIDER: S-EPMC7844988 | biostudies-literature | 2021 Jan

REPOSITORIES: biostudies-literature