Diamond-Blackfan anemia (DBA) is one of the most common inherited causes of bone
marrow failure in children. DBA typically presents with isolated erythroid hypoplasia and …

Mice with a constitutive increase in p53 activity exhibited features of dyskeratosis congenita
(DC), a bone marrow failure syndrome (BMFS) caused by defective telomere maintenance …

This study lays the groundwork for future lentivirus-mediated gene therapy in patients with
Diamond Blackfan anemia (DBA) caused by mutations in ribosomal protein S19 (RPS19) …

Recent advances in in-depth data-independent acquisition proteomic analysis have
enabled comprehensive quantitative analysis of> 10,000 proteins. Herein, an integrated …

Diverse acute and chronic injuries induce damage responses in the gastrointestinal (GI)
system, and numerous cell types in the gastrointestinal tract demonstrate remarkable …

Genetic manipulation of hematopoietic stem cells (HSCs) is being developed as a
therapeutic strategy for several inherited disorders. This field is rapidly evolving with several …

The clinical potential that stems from the discovery of DNA's double helix in 1953, and the
subsequent genomic knowledge about health and disease, is now beginning to be realized …

Diamond-Blackfan anemia syndrome (DBAS) is an inherited bone marrow failure disorder
that typically presents in infancy as hypoplastic anemia and developmental abnormalities in …

This article was submitted to Hematologic Malignancies, a section of the journal Frontiers in
Oncology RECEIVED 26 November 2022 Page 65 This article was submitted to Hematologic …

The neighbouring genomic positions of Nprl3 and a-globin, which share a topologically
associated domain, have been maintained for over 500 million years. Nprl3 contains 4 of the …