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Hence, our recent identification of HLY78 as a novel Wnt activator, may provide new chances for the circumstances where activation of Wnt signaling is desired, such as for the patients with bone marrow failure or undergoing post-HSC transplantation (Wang et al., 2013).
Bone marrow failure or spontaneous and sporadic anemia has been reported in TR-knockout mice [50], but not in TERT-knockout mice, although further characterizations of TERT are required in the mouse.
This finding is distinct from the known regulatory role of apoptosis in bone marrow failure or anemia caused by ribosome disorders.
All were judged as requiring treatment (progressive disease over at least 3 months, critical organ involvement, bone marrow failure or 'B' symptoms).
The patient, however, had no symptoms of bone marrow failure or malignancies when diagnosed, so bi-allelic inactivation of RAD51C has been provisionally associated with a FA-like disorder.
Hence, we believe that we are in need for better treatment strategies to avoid unnecessary tissue damage and inflammatory responses in tumor patients as well as in patients presenting with premalignant conditions such as bone marrow failure or viral hepatitis.
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DKC is considered a syndrome of premature aging with death occurring at a median age of 16 years and a maximum of 50, usually from bone marrow failure, cancer or pulmonary disease[14].
Currently, preimplantation genetic diagnosis (PGD) is used to obtain a human leukocyte antigen (HLA -matched cHLA -matchednor for childblood HSC to trassplant andonore a sibling with bone marrow forlure syndromes or other hematologicorddiseases (bloodrai et al., 2004; Grewal et al., 2004).
Haploidentical transplantation opens the possibility to offer this treatment to a large number of patients with an otherwise incurable disease, such as some hematologic or oncologic malignancies, inborn or acquired bone marrow failure syndromes, hemoglobinopathies, immunodeficiencies, or other genetic diseases.
Myelofibrosis is associated with a much poorer prognosis and significant disease burden affecting quality of life, usually from progressive bone marrow failure, extramedullary haemopoiesis or transformation to aggressive leukaemia.
Although it is not clear whether the association between ribosomal defects and bone marrow failure is coincidental or causal, the various theories supporting this association have been examined in comprehensive reviews [16], [17].
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